Trial Outcomes & Findings for A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders (NCT NCT02246218)
NCT ID: NCT02246218
Last Updated: 2024-07-01
Results Overview
The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
27 participants
Primary outcome timeframe
Up to Day 4
Results posted on
2024-07-01
Participant Flow
A total of 27 participants were enrolled; 1 participant was not dosed and is not included in any analysis.
Participant milestones
| Measure |
RAVICTI: Age 2 Months to < 2 Years
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
RAVICTI: Age 0 to < 2 Months
Participants age 0 to \< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
16
|
|
Overall Study
COMPLETED
|
6
|
10
|
|
Overall Study
NOT COMPLETED
|
4
|
6
|
Reasons for withdrawal
| Measure |
RAVICTI: Age 2 Months to < 2 Years
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
RAVICTI: Age 0 to < 2 Months
Participants age 0 to \< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|---|
|
Overall Study
Withdrawal by Parent/Guardian
|
0
|
1
|
|
Overall Study
Stopping Rule: Liver Transplant
|
2
|
4
|
|
Overall Study
Adverse Event
|
1
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
Baseline Characteristics
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Baseline characteristics by cohort
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
RAVICTI: Age 0 to <2 Months
n=16 Participants
Participants age 0 to \< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
Total
n=26 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
9.87 months
STANDARD_DEVIATION 5.529 • n=5 Participants
|
0.83 months
STANDARD_DEVIATION 0.697 • n=7 Participants
|
4.31 months
STANDARD_DEVIATION 5.606 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to Day 4Population: Safety Population: all enrolled participants who received any amount of study medication.
The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 2 Months to <2 Years Participants
|
100 percentage of participants
|
PRIMARY outcome
Timeframe: Up to Day 4Population: Safety Population: all enrolled participants who received any amount of study medication.
The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants \< 2 months of age, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 0 Months to <2 Months Participants
|
100 percentage of participants
|
SECONDARY outcome
Timeframe: Day 8 through up to Month 6Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
HAC is defined having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension is calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=9 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Rate of Hyperammonemic Crises (HACs): Cohort of 2 Months to <2 Years Participants
|
0.005 HACs per half-year of patient exposure
|
SECONDARY outcome
Timeframe: From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months).Population: Safety Population: all enrolled participants who received any amount of study medication.
An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants
≥ 1 TEAE
|
10 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants
≥ 1 Related TEAE
|
4 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants
≥ 1 Serious TEAE
|
6 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants
≥ 1 Serious Related TEAE
|
0 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants
Fatal Outcome TEAE
|
1 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants
≥ 1 TEAE Leading to Study Discontinuation
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
122.43 µmol/L
Standard Deviation 118.620
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Day 7 change from Baseline
|
-54.50 µmol/L
Standard Deviation 92.626
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
7.80 µmol/L
Standard Deviation 25.646
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
-16.33 µmol/L
Standard Deviation 39.209
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
-13.00 µmol/L
Standard Deviation 39.590
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
0.25 µmol/L
Standard Deviation 13.426
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
-2.20 µmol/L
Standard Deviation 34.463
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
30.80 µmol/L
Standard Deviation 17.092
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
22.20 µmol/L
Standard Deviation 23.506
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
39.00 µmol/L
Standard Deviation NA
1 participant analyzed
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
48.00 µmol/L
Standard Deviation 53.740
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
750.43 µmol/L
Standard Deviation 309.000
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Day 7 change from Baseline
|
-184.33 µmol/L
Standard Deviation 168.657
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
-174.60 µmol/L
Standard Deviation 318.249
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
-374.00 µmol/L
Standard Deviation 425.903
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
-252.75 µmol/L
Standard Deviation 323.852
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
-370.25 µmol/L
Standard Deviation 377.222
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
-113.20 µmol/L
Standard Deviation 519.710
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
-446.53 µmol/L
Standard Deviation 360.457
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
-450.50 µmol/L
Standard Deviation 386.699
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
-149.00 µmol/L
Standard Deviation NA
1 participant with an assessment
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
195.00 µmol/L
Standard Deviation 554.372
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
872.86 µmol/L
Standard Deviation 381.005
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Day 7 change from Baseline
|
-238.83 µmol/L
Standard Deviation 203.567
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
-166.80 µmol/L
Standard Deviation 332.421
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
-390.33 µmol/L
Standard Deviation 462.292
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
-265.75 µmol/L
Standard Deviation 339.015
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
-370.00 µmol/L
Standard Deviation 379.884
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
-115.40 µmol/L
Standard Deviation 546.796
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
-415.73 µmol/L
Standard Deviation 365.419
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
-428.30 µmol/L
Standard Deviation 404.351
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
-110.00 µmol/L
Standard Deviation NA
1 participant with an assessment
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
243.00 µmol/L
Standard Deviation 608.112
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
54.86 µmol/L
Standard Deviation 19.497
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Day 7 change from Baseline
|
2.67 µmol/L
Standard Deviation 19.159
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
4.20 µmol/L
Standard Deviation 45.861
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
-25.67 µmol/L
Standard Deviation 16.743
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
-20.25 µmol/L
Standard Deviation 20.353
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
-20.00 µmol/L
Standard Deviation 36.341
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
-16.40 µmol/L
Standard Deviation 12.137
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
-6.73 µmol/L
Standard Deviation 13.342
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
-13.33 µmol/L
Standard Deviation 15.885
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
-18.00 µmol/L
Standard Deviation NA
1 participant with an assessment
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
1.50 µmol/L
Standard Deviation 10.607
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
90.86 µmol/L
Standard Deviation 29.249
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Day 7 change from Baseline
|
-0.83 µmol/L
Standard Deviation 32.762
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
9.80 µmol/L
Standard Deviation 65.975
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
-33.00 µmol/L
Standard Deviation 25.239
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
-31.25 µmol/L
Standard Deviation 24.771
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
-39.50 µmol/L
Standard Deviation 61.136
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
-25.40 µmol/L
Standard Deviation 13.594
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
-19.13 µmol/L
Standard Deviation 39.322
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
-34.37 µmol/L
Standard Deviation 23.283
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
-40.00 µmol/L
Standard Deviation NA
1 participant with an assessment
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
-1.50 µmol/L
Standard Deviation 31.820
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
-46.00 µmol/L
Standard Deviation NA
1 participant with an assessment
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
171.43 µmol/L
Standard Deviation 42.887
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Day 7 change from Baseline
|
4.00 µmol/L
Standard Deviation 24.133
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
40.60 µmol/L
Standard Deviation 90.057
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
-27.33 µmol/L
Standard Deviation 37.018
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
-31.50 µmol/L
Standard Deviation 29.760
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
-56.00 µmol/L
Standard Deviation 75.939
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
-21.60 µmol/L
Standard Deviation 18.202
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
-11.90 µmol/L
Standard Deviation 71.753
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
-48.87 µmol/L
Standard Deviation 51.644
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
5.00 µmol/L
Standard Deviation 55.154
|
SECONDARY outcome
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
0.8107 z-score
Standard Deviation 2.17017
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 1 change from Baseline
|
-0.2385 z-score
Standard Deviation 0.77830
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
-0.0249 z-score
Standard Deviation 0.74861
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
0.1815 z-score
Standard Deviation 0.86056
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
0.4434 z-score
Standard Deviation 0.94854
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
0.1484 z-score
Standard Deviation 0.76293
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
0.2497 z-score
Standard Deviation 0.80923
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
0.6407 z-score
Standard Deviation 0.98695
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
0.4164 z-score
Standard Deviation 0.80674
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
-0.2997 z-score
Standard Deviation 0.16959
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 18 change from Baseline
|
-0.2038 z-score
Standard Deviation NA
1 participant with an assessment
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
0.5581 z-score
Standard Deviation 1.23993
|
SECONDARY outcome
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Baseline
|
0.7143 z-score
Standard Deviation 2.14922
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 1 change from Baseline
|
-0.2105 z-score
Standard Deviation 0.74135
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 2 change from Baseline
|
-0.0704 z-score
Standard Deviation 0.70393
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 3 change from Baseline
|
0.1065 z-score
Standard Deviation 0.70165
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 4 change from Baseline
|
0.3365 z-score
Standard Deviation 0.77672
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 5 change from Baseline
|
0.1043 z-score
Standard Deviation 0.56747
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 6 change from Baseline
|
0.1842 z-score
Standard Deviation 0.62205
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 9 change from Baseline
|
0.4875 z-score
Standard Deviation 0.86137
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 12 change from Baseline
|
0.2944 z-score
Standard Deviation 0.75133
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 15 change from Baseline
|
-0.3661 z-score
Standard Deviation 0.00932
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 18 change from Baseline
|
-0.2214 z-score
Standard Deviation NA
1 participant with assessment
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants
Month 24 change from Baseline
|
0.4310 z-score
Standard Deviation 1.13140
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: Pharmacokinetic (PK) Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma Phenylbutyrate/Phenylbutyric Acid (PBA) Maximum Plasma Concentration (Cmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
42.44 μg/mL
Standard Deviation 36.715
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PBA Minimum Plasma Concentration (Cmin) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
1.697 μg/mL
Standard Deviation 2.254
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PBA Area Under the Curve From Time Zero to the Time of Last Quantifiable Concentration (AUC[0-last]) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
280.936 μg*hr/mL
Standard Deviation 293.553
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PBA Time to Cmax (Tmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
8.383 hours
Standard Deviation 4.564
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma Phenylacetate/Phenylacetic Acid (PAA) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
36.52 μg/mL
Standard Deviation 31.784
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
4.197 μg/mL
Standard Deviation 6.434
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
246.126 μg*hr/mL
Standard Deviation 238.547
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
7.422 hours
Standard Deviation 7.351
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma Phenylacetylglutamine (PAGN) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
62.45 μg/mL
Standard Deviation 27.281
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
20.62 μg/mL
Standard Deviation 14.529
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
583.835 μg*hr/mL
Standard Deviation 285.241
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
|
6.573 hours
Standard Deviation 7.181
|
SECONDARY outcome
Timeframe: Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
Hour 0
|
3273 μg/mL
Standard Deviation 1993
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
0.5 to 1.5 hours
|
4140 μg/mL
Standard Deviation 4399
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
1.5 to 2.5 hours
|
3145 μg/mL
Standard Deviation 5045
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
4 to 6 hours
|
5202 μg/mL
Standard Deviation 4547
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
7.5 to 8.5 hours
|
3950 μg/mL
Standard Deviation 3068
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
12 to 24 hours
|
7561 μg/mL
Standard Deviation 6956
|
SECONDARY outcome
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, End of Trial (up to Month 18)Population: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 9
|
9357 μg/mL
Standard Deviation 7286
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Day 7
|
8859 μg/mL
Standard Deviation 10500
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 1
|
6274 μg/mL
Standard Deviation 4802
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 2
|
7386 μg/mL
Standard Deviation 6419
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 3
|
11456 μg/mL
Standard Deviation 14471
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 4
|
21416 μg/mL
Standard Deviation 33695
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 5
|
6129 μg/mL
Standard Deviation 8024
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 6
|
5347 μg/mL
Standard Deviation 3153
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 12
|
2580 μg/mL
Standard Deviation 286
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 15
|
6400 μg/mL
Standard Deviation NA
1 participant assessed
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 18
|
5250 μg/mL
Standard Deviation NA
1 participant assessed
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
End of trial
|
25333 μg/mL
Standard Deviation 21324
|
SECONDARY outcome
Timeframe: Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
Hour 0
|
18.78 μg/mL
Standard Deviation 27.33
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
0.5 to 1.5 hours
|
6.50 μg/mL
Standard Deviation 3.39
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
1.5 to 2.5 hours
|
7.29 μg/mL
Standard Deviation 4.10
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
4 to 6 hours
|
2.60 μg/mL
Standard Deviation 2.18
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
7.5 to 8.5 hours
|
4.48 μg/mL
Standard Deviation 4.38
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants
12 to 24 hours
|
4.31 μg/mL
Standard Deviation 2.33
|
SECONDARY outcome
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, Month 18, End of Trial (up to Month 18)Population: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Day 7
|
5.82 μg/mL
Standard Deviation 2.21
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 1
|
4.44 μg/mL
Standard Deviation 4.61
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 2
|
3.69 μg/mL
Standard Deviation 1.82
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 3
|
4.65 μg/mL
Standard Deviation 0.49
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 4
|
7.14 μg/mL
Standard Deviation NA
1 participant assessed
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 5
|
3.27 μg/mL
Standard Deviation 1.96
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 6
|
1.59 μg/mL
Standard Deviation NA
1 participant assessed
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 9
|
4.10 μg/mL
Standard Deviation 1.65
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 15
|
2.04 μg/mL
Standard Deviation NA
1 participant assessed
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
Month 18
|
1.64 μg/mL
Standard Deviation NA
1 participant assessed
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants
End of trial
|
7.0 μg/mL
Standard Deviation 5.16
|
SECONDARY outcome
Timeframe: Day 8 through up to Month 6Population: Safety Population: all enrolled participants who received any amount of study medication.
HAC is defined as having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension was calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Rate of HACs: Cohort of 0 Months to <2 Months Participants
|
0.003 HACs per half-year of patient exposure
|
SECONDARY outcome
Timeframe: From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 10.67 [6.142] months).Population: Safety Population: all enrolled participants who received any amount of study medication.
An AE is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants
≥ 1 TEAE
|
16 Participants
|
|
Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants
≥ 1 Related TEAE
|
10 Participants
|
|
Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants
≥ 1 Serious TEAE
|
11 Participants
|
|
Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants
≥ 1 Serious Related TEAE
|
0 Participants
|
|
Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants
Fatal Outcome TEAE
|
0 Participants
|
|
Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants
≥ 1 TEAE Leading to Study Discontinuation
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
84.97 μmol/L
Standard Deviation 52.086
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Day 7 change from Baseline
|
26.81 μmol/L
Standard Deviation 76.417
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
25.16 μmol/L
Standard Deviation 64.474
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
50.05 μmol/L
Standard Deviation 73.988
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
18.77 μmol/L
Standard Deviation 67.561
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
57.43 μmol/L
Standard Deviation 98.550
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
43.65 μmol/L
Standard Deviation 140.985
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
33.41 μmol/L
Standard Deviation 150.273
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
8.75 μmol/L
Standard Deviation 97.329
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
25.75 μmol/L
Standard Deviation 62.660
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
2.50 μmol/L
Standard Deviation 21.920
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
16.10 μmol/L
Standard Deviation 95.461
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
508.83 μmol/L
Standard Deviation 337.175
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Day 7 change from Baseline
|
21.04 μmol/L
Standard Deviation 260.500
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
-27.62 μmol/L
Standard Deviation 379.796
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
-15.09 μmol/L
Standard Deviation 352.471
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
-113.98 μmol/L
Standard Deviation 230.855
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
-99.82 μmol/L
Standard Deviation 305.674
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
-138.16 μmol/L
Standard Deviation 349.269
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
-56.08 μmol/L
Standard Deviation 269.288
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
-181.50 μmol/L
Standard Deviation 118.604
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
-103.75 μmol/L
Standard Deviation 328.583
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
-184.00 μmol/L
Standard Deviation 80.610
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
-219.93 μmol/L
Standard Deviation 279.815
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
593.80 μmol/L
Standard Deviation 333.657
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Day 7 change from Baseline
|
47.85 μmol/L
Standard Deviation 230.343
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
-2.46 μmol/L
Standard Deviation 402.919
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
34.96 μmol/L
Standard Deviation 371.522
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
-95.21 μmol/L
Standard Deviation 238.136
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
-42.39 μmol/L
Standard Deviation 288.782
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
-94.51 μmol/L
Standard Deviation 297.378
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
-22.66 μmol/L
Standard Deviation 318.034
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
-172.75 μmol/L
Standard Deviation 210.202
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
-78.00 μmol/L
Standard Deviation 297.410
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
-181.50 μmol/L
Standard Deviation 102.530
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
-203.83 μmol/L
Standard Deviation 255.810
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
142.68 μmol/L
Standard Deviation 222.694
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Day 7 change from Baseline
|
-49.09 μmol/L
Standard Deviation 198.730
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
-1.62 μmol/L
Standard Deviation 233.754
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
-20.46 μmol/L
Standard Deviation 216.885
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
-67.32 μmol/L
Standard Deviation 173.564
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
-75.45 μmol/L
Standard Deviation 221.845
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
-35.94 μmol/L
Standard Deviation 78.367
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
-73.09 μmol/L
Standard Deviation 245.051
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
-178.50 μmol/L
Standard Deviation 335.538
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
-139.50 μmol/L
Standard Deviation 359.287
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
1.00 μmol/L
Standard Deviation 45.255
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
-55.31 μmol/L
Standard Deviation 217.209
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
133.67 μmol/L
Standard Deviation 253.829
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Day 7 change from Baseline
|
-81.91 μmol/L
Standard Deviation 273.127
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
-60.80 μmol/L
Standard Deviation 278.083
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
-51.66 μmol/L
Standard Deviation 269.558
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
-82.82 μmol/L
Standard Deviation 224.269
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
-118.55 μmol/L
Standard Deviation 311.125
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
-11.85 μmol/L
Standard Deviation 40.693
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
-115.09 μmol/L
Standard Deviation 338.186
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
-249.50 μmol/L
Standard Deviation 483.776
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
-195.75 μmol/L
Standard Deviation 496.313
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
6.00 μmol/L
Standard Deviation 62.225
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
-82.54 μmol/L
Standard Deviation 228.107
|
SECONDARY outcome
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
-238.25 μmol/L
Standard Deviation 506.435
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
-137.00 μmol/L
Standard Deviation 519.042
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
181.49 μmol/L
Standard Deviation 257.948
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Day 7 change from Baseline
|
-63.96 μmol/L
Standard Deviation 290.312
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
-39.04 μmol/L
Standard Deviation 297.634
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
-23.86 μmol/L
Standard Deviation 290.128
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
-74.41 μmol/L
Standard Deviation 233.538
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
-98.67 μmol/L
Standard Deviation 326.300
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
2.64 μmol/L
Standard Deviation 77.843
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
-90.40 μmol/L
Standard Deviation 355.316
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
38.00 μmol/L
Standard Deviation 67.882
|
|
Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
-72.78 μmol/L
Standard Deviation 258.710
|
SECONDARY outcome
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
-0.0544 z-score
Standard Deviation 1.26821
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 1 change from Baseline
|
-0.2158 z-score
Standard Deviation 1.35960
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
-0.2598 z-score
Standard Deviation 1.19544
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
-0.1617 z-score
Standard Deviation 1.02572
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
-0.0264 z-score
Standard Deviation 1.68215
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
0.0828 z-score
Standard Deviation 1.14206
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
0.0136 z-score
Standard Deviation 1.72106
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
0.4614 z-score
Standard Deviation 1.25343
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
0.6646 z-score
Standard Deviation 0.95334
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
0.6830 z-score
Standard Deviation 0.55703
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
0.3308 z-score
Standard Deviation 0.27572
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
0.7743 z-score
Standard Deviation 0.64962
|
SECONDARY outcome
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24Population: Safety Population: all enrolled participants who received any amount of study medication and had an assessment.
To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Baseline
|
-0.1980 z-score
Standard Deviation 2.11774
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 1 change from Baseline
|
0.2336 z-score
Standard Deviation 1.95603
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 2 change from Baseline
|
0.2006 z-score
Standard Deviation 1.72135
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 3 change from Baseline
|
0.2684 z-score
Standard Deviation 1.37517
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 4 change from Baseline
|
0.2372 z-score
Standard Deviation 2.30831
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 5 change from Baseline
|
0.1810 z-score
Standard Deviation 1.70024
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 6 change from Baseline
|
0.2902 z-score
Standard Deviation 2.05956
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 9 change from Baseline
|
0.1679 z-score
Standard Deviation 1.32407
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 12 change from Baseline
|
0.1308 z-score
Standard Deviation 0.50371
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 15 change from Baseline
|
0.1595 z-score
Standard Deviation 0.75833
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 18 change from Baseline
|
0.1050 z-score
Standard Deviation 0.73521
|
|
Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants
Month 24 change from Baseline
|
0.7341 z-score
Standard Deviation 1.35582
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PBA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
46.2 μg/mL
Standard Deviation 49.8
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=7 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PBA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
4.8 μg/mL
Standard Deviation 4.2
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PBA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
374.53 μg*hr/mL
Standard Deviation 390.48
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PBA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
9.39 hours
Standard Deviation 7.41
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
115.3 μg/mL
Standard Deviation 102.0
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
98.98 μg/mL
Standard Deviation 122.07
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
1321.18 μg*hr/mL
Standard Deviation 1220.52
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
9.85 hours
Standard Deviation 9.26
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAGN Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
102.1 μg/mL
Standard Deviation 48.6
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=13 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
69.39 μg/mL
Standard Deviation 54.03
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
1384.12 μg*hr/mL
Standard Deviation 1141.03
|
SECONDARY outcome
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
|
11.72 hours
Standard Deviation 8.24
|
SECONDARY outcome
Timeframe: Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
Hour 0
|
3530.43 μg/mL
Standard Deviation 3600.4
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
0.5 to 1.5 hours
|
1828 μg/mL
Standard Deviation 2862
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
1.5 to 2.5 hours
|
1746 μg/mL
Standard Deviation 1464
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
4 to 6 hours
|
2260 μg/mL
Standard Deviation 1472
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
7.5 to 8.5 hours
|
3530.43 μg/mL
Standard Deviation 3600.4
|
|
Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
12 to 24 hours
|
4404 μg/mL
Standard Deviation 3766
|
SECONDARY outcome
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, End of Trial (up to Month 15)Population: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Day 7
|
4643 μg/mL
Standard Deviation 2506
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 1
|
4517 μg/mL
Standard Deviation 2485
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 2
|
4116 μg/mL
Standard Deviation 3137
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 3
|
7037 μg/mL
Standard Deviation 4493
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 4
|
2826 μg/mL
Standard Deviation 1543
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 5
|
6973 μg/mL
Standard Deviation 3682
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 6
|
5883 μg/mL
Standard Deviation 3128
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 9
|
7006 μg/mL
Standard Deviation 4289
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 12
|
5847 μg/mL
Standard Deviation 2992
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 15
|
3915 μg/mL
Standard Deviation 2584
|
|
Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
End of trial
|
6939 μg/mL
Standard Deviation 6581
|
SECONDARY outcome
Timeframe: Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HACPopulation: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
Hour 0
|
11.1 μg/mL
Standard Deviation 10.4
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
0.5 to 1.5 hours
|
46.2 μg/mL
Standard Deviation 58.6
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
1.5 to 2.5 hours
|
62.5 μg/mL
Standard Deviation 42.8
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
4 to 6 hours
|
34.6 μg/mL
Standard Deviation 56.2
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
7.5 to 8.5 hours
|
22.8 μg/mL
Standard Deviation 25.3
|
|
Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants
12 to 24 hours
|
35.2 μg/mL
Standard Deviation 51.0
|
SECONDARY outcome
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, End of Trial (up to Month 15)Population: PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.
Outcome measures
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=16 Participants
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Day 7
|
23.7 μg/mL
Standard Deviation 31.2
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 1
|
14.6 μg/mL
Standard Deviation 17.2
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 2
|
12.3 μg/mL
Standard Deviation 13.3
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 3
|
14.4 μg/mL
Standard Deviation 10.7
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 4
|
6.4 μg/mL
Standard Deviation 7.1
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 5
|
13.2 μg/mL
Standard Deviation 14.0
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 6
|
5.5 μg/mL
Standard Deviation NA
1 participant assessed
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 9
|
11.8 μg/mL
Standard Deviation 8.5
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 12
|
6.0 μg/mL
Standard Deviation 3.8
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
Month 15
|
4.9 μg/mL
Standard Deviation 0.3
|
|
Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants
End of trial
|
11.6 μg/mL
Standard Deviation 9.0
|
Adverse Events
RAVICTI: Age 2 Months to < 2 Years
Serious events: 6 serious events
Other events: 8 other events
Deaths: 1 deaths
RAVICTI: Age 0 to <2 Months
Serious events: 11 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 participants at risk
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
RAVICTI: Age 0 to <2 Months
n=16 participants at risk
Participants age 0 to \< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|---|
|
Investigations
Ammonia increased
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Cardiac disorders
Cyanosis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Apnoeic attack
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Status asthmaticus
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
General disorders
Pyrexia
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Pneumatosis intestinalis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Hyperammonaemia
|
30.0%
3/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
31.2%
5/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Feeding disorder
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Croup infectious
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Gastroenteritis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Urinary tract infection
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Viral infection
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Influenza
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Peritonitis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Rhinovirus infection
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Bacteraemia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Cellulitis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Device related infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Medical device site infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Meningitis bacterial
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Tracheitis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
Other adverse events
| Measure |
RAVICTI: Age 2 Months to < 2 Years
n=10 participants at risk
Participants age 2 months to \< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
RAVICTI: Age 0 to <2 Months
n=16 participants at risk
Participants age 0 to \< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
|
|---|---|---|
|
Vascular disorders
Venous thrombosis limb
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Immune system disorders
Seasonal allergy
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
General disorders
Pyrexia
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
General disorders
Catheter site rash
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
General disorders
Device occlusion
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
General disorders
Drug withdrawal syndrome
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
General disorders
Medical device site haemorrhage
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Psychiatric disorders
Agitation
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Psychiatric disorders
Irritability
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Injury, poisoning and procedural complications
Stoma site reaction
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Amino acid level decreased
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Ammonia increased
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Carbon dioxide decreased
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Hepatic enzyme increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Amino acid level increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Anion gap increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Blood bicarbonate decreased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Blood urea decreased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Body height below normal
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Platelet count increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Transaminases increased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Investigations
Weight decreased
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Congenital, familial and genetic disorders
Dacryostenosis congenital
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Congenital, familial and genetic disorders
Plagiocephaly
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Anaemia
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
18.8%
3/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Thrombocytosis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Lymphocytosis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Blood and lymphatic system disorders
Microcytic anaemia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
25.0%
4/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Apnoea
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Use of accessory respiratory muscles
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Nervous system disorders
Gross motor delay
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Nervous system disorders
Tremor
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Eye disorders
Eye discharge
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Eye disorders
Ocular hyperaemia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Ear and labyrinth disorders
Excessive cerumen production
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Diarrhoea
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
31.2%
5/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Vomiting
|
40.0%
4/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
31.2%
5/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Teething
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
18.8%
3/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Constipation
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
37.5%
6/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
18.8%
3/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Gastrointestinal disorders
Post-tussive vomiting
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Hepatobiliary disorders
Hepatic calcification
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Renal and urinary disorders
Vesicoureteric reflux
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Skin and subcutaneous tissue disorders
Nail ridging
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Skin and subcutaneous tissue disorders
Rash
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
31.2%
5/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
37.5%
6/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Skin and subcutaneous tissue disorders
Red man syndrome
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Skin and subcutaneous tissue disorders
Seborrhoeic dermatitis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Musculoskeletal and connective tissue disorders
Torticollis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Hypophagia
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Hyperammonaemia
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Feeding disorder of infancy or early childhood
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Metabolism and nutrition disorders
Protein deficiency
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Gastroenteritis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Upper respiratory tract infection
|
40.0%
4/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
31.2%
5/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Urinary tract infection
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Viral infection
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Nasopharyngitis
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
25.0%
4/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Croup infectious
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Otitis media
|
20.0%
2/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Conjunctivitis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Pharyngitis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Pneumonia
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
0.00%
0/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Sinusitis
|
10.0%
1/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Ear infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
18.8%
3/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Oral candidiasis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
12.5%
2/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Angular cheilitis
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Candida infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Gastrointestinal viral infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Otitis media acute
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
|
Infections and infestations
Rhinovirus infection
|
0.00%
0/10 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
6.2%
1/16 • From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).
TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.
- Publication restrictions are in place
Restriction type: OTHER