Trial Outcomes & Findings for A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia (NCT NCT03519711)
NCT ID: NCT03519711
Last Updated: 2023-11-14
Results Overview
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. TEAEs were defined as AEs that commenced or worsened after the first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
8 participants
Primary outcome timeframe
From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
Results posted on
2023-11-14
Participant Flow
Participant milestones
| Measure |
Cohort 1: PTC923 2.5 and 10 mg/kg/Day
Participants received PTC923 (CNSA-001) suspension 2.5 milligrams (mg)/kilogram (kg)/day (dose divided in 2 for twice daily administration) orally for 7 days in Period 1, underwent a 3 (±1) day washout period, then escalated to a dose of 10 mg/kg/day (dose divided in 2 for twice daily administration) administered orally for 7 days in Period 2 (14 days total treatment).
|
Cohort 2: PTC923 5 and 20 mg/kg/Day
Participants received PTC923 suspension 5 mg/kg/day (dose divided in 2 for twice daily administration) orally for 7 days in Period 1, underwent a 3 (±1) day washout period, then escalated to a dose of 20 mg/kg/day (dose divided in 2 for twice daily administration) administered orally for 7 days in Period 2 (14 days total treatment).
|
|---|---|---|
|
Treatment Period 1 (7 Days)
STARTED
|
4
|
4
|
|
Treatment Period 1 (7 Days)
Received at Least 1 Dose of Study Drug
|
4
|
4
|
|
Treatment Period 1 (7 Days)
COMPLETED
|
4
|
4
|
|
Treatment Period 1 (7 Days)
NOT COMPLETED
|
0
|
0
|
|
Treatment Period 2 (7 Days)
STARTED
|
4
|
4
|
|
Treatment Period 2 (7 Days)
COMPLETED
|
4
|
4
|
|
Treatment Period 2 (7 Days)
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
Baseline characteristics by cohort
| Measure |
Cohort 1: PTC923 2.5 and 10 mg/kg/Day
n=4 Participants
Participants received PTC923 suspension 2.5 mg/kg/day (dose divided in 2 for twice daily administration) orally for 7 days in Period 1, underwent a 3 (±1) day washout period, then escalated to a dose of 10 mg/kg/day (dose divided in 2 for twice daily administration) administered orally for 7 days in Period 2 (14 days total treatment).
|
Cohort 2: PTC923 5 and 20 mg/kg/Day
n=4 Participants
Participants received PTC923 suspension 5 mg/kg/day (dose divided in 2 for twice daily administration) orally for 7 days in Period 1, underwent a 3 (±1) day washout period, then escalated to a dose of 20 mg/kg/day (dose divided in 2 for twice daily administration) administered orally for 7 days in Period 2 (14 days total treatment).
|
Total
n=8 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
11.75 years
STANDARD_DEVIATION 5.123 • n=5 Participants
|
11.55 years
STANDARD_DEVIATION 8.786 • n=7 Participants
|
11.65 years
STANDARD_DEVIATION 6.659 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)Population: The Safety population included all randomized participants who received any amount of study drug.
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. TEAEs were defined as AEs that commenced or worsened after the first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=4 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=4 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
|
2 Participants
|
2 Participants
|
4 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Day 1 (pre-dose, within 30 minutes of dosing), 0.5, 1, 2, 4, 6, 8 hours postdose (prior to Day 1 evening dose), and 24 hours postdose (prior to Day 2 morning dose)Population: The PK population included all participants who received at least 1 dose of study drug and had at least 1 quantifiable post-dose blood sample collected for analysis of PTC923, BH4, Phe, or Tyr concentrations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=3 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=3 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of PTC923 and Tetrahydrobiopterin (BH4)
PTC923
|
—
|
1.110 nanograms (ng)/milliliter (mL)
Standard Deviation 0.4031
|
0.533 nanograms (ng)/milliliter (mL)
Standard Deviation 0.5033
|
2.120 nanograms (ng)/milliliter (mL)
Standard Deviation 0.5292
|
|
Maximum Observed Plasma Concentration (Cmax) of PTC923 and Tetrahydrobiopterin (BH4)
BH4
|
19.455 nanograms (ng)/milliliter (mL)
Standard Deviation 19.3731
|
109.375 nanograms (ng)/milliliter (mL)
Standard Deviation 58.9440
|
48.200 nanograms (ng)/milliliter (mL)
Standard Deviation 20.1570
|
275.030 nanograms (ng)/milliliter (mL)
Standard Deviation 42.7941
|
SECONDARY outcome
Timeframe: Day 1 (pre-dose, within 30 minutes of dosing), 0.5, 1, 2, 4, 6, 8 hours postdose (prior to Day 1 evening dose), 24 hours postdose (prior to Day 2 morning dose), 72 hours postdose (Day 4), on Day 7, and at the end of study (48 hours after last dose)Population: The PK population included all participants who received at least 1 dose of study drug and had at least 1 quantifiable post-dose blood sample collected for analysis of PTC923, BH4, Phe, or Tyr concentrations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=3 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=3 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Cmax of Phenylalanine (Phe) and Tyrosine (Tyr)
Tyr
|
157.15 micromoles (µmol)/liter (L)
Standard Deviation 31.229
|
152.98 micromoles (µmol)/liter (L)
Standard Deviation 22.314
|
173.40 micromoles (µmol)/liter (L)
Standard Deviation 42.981
|
178.13 micromoles (µmol)/liter (L)
Standard Deviation 48.925
|
|
Cmax of Phenylalanine (Phe) and Tyrosine (Tyr)
Phe
|
939.35 micromoles (µmol)/liter (L)
Standard Deviation 1059.225
|
725.98 micromoles (µmol)/liter (L)
Standard Deviation 883.411
|
1420.30 micromoles (µmol)/liter (L)
Standard Deviation 487.712
|
991.63 micromoles (µmol)/liter (L)
Standard Deviation 555.802
|
SECONDARY outcome
Timeframe: Day 1 (pre-dose, within 30 minutes of dosing), 0.5, 1, 2, 4, 6, 8 hours postdose (prior to Day 1 evening dose), and 24 hours postdose (prior to Day 2 morning dose)Population: The PK population included all participants who received at least 1 dose of study drug and had at least 1 quantifiable post-dose blood sample collected for analysis of PTC923, BH4, Phe, or Tyr concentrations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=3 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=3 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Area Under the Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of PTC923 and BH4
BH4
|
72.826 hours*ng/mL
Standard Deviation 62.8038
|
457.330 hours*ng/mL
Standard Deviation 204.2517
|
221.358 hours*ng/mL
Standard Deviation 82.6201
|
1158.337 hours*ng/mL
Standard Deviation 227.0332
|
|
Area Under the Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of PTC923 and BH4
PTC923
|
—
|
2.532 hours*ng/mL
Standard Deviation 2.2370
|
—
|
5.605 hours*ng/mL
Standard Deviation 1.0517
|
SECONDARY outcome
Timeframe: Day 1 (pre-dose, within 30 minutes of dosing), 0.5, 1, 2, 4, 6, 8 hours postdose (prior to Day 1 evening dose), 24 hours postdose (prior to Day 2 morning dose), 72 hours postdose (Day 4), on Day 7, and at the end of study (48 hours after last dose)Population: The PK population included all participants who received at least 1 dose of study drug and had at least 1 quantifiable post-dose blood sample collected for analysis of PTC923, BH4, Phe, or Tyr concentrations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=3 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=3 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
AUC0-last of Phe and Tyr
Tyr
|
883.337 hours*µmol/L
Standard Deviation 65.2122
|
836.526 hours*µmol/L
Standard Deviation 143.4899
|
1037.085 hours*µmol/L
Standard Deviation 346.7325
|
1023.856 hours*µmol/L
Standard Deviation 368.7586
|
|
AUC0-last of Phe and Tyr
Phe
|
3636.030 hours*µmol/L
Standard Deviation 4275.9593
|
1877.091 hours*µmol/L
Standard Deviation 1633.8636
|
3655.897 hours*µmol/L
Standard Deviation 1239.8499
|
2086.787 hours*µmol/L
Standard Deviation 871.7262
|
SECONDARY outcome
Timeframe: Day 1 (pre-dose, within 30 minutes of dosing), 0.5, 1, 2, 4, 6, 8 hours postdose (prior to Day 1 evening dose), and 24 hours postdose (prior to Day 2 morning dose)Population: The PK population included all participants who received at least 1 dose of study drug and had at least 1 quantifiable post-dose blood sample collected for analysis of PTC923, BH4, Phe, or Tyr concentrations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=3 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=3 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Time to Reach Cmax (Tmax) of PTC923 and BH4
PTC923
|
—
|
1.000 hours
Interval 0.5 to 2.03
|
1.000 hours
Interval 0.0 to 1.02
|
1.000 hours
Interval 0.98 to 2.0
|
|
Time to Reach Cmax (Tmax) of PTC923 and BH4
BH4
|
4.017 hours
Interval 3.87 to 5.88
|
3.050 hours
Interval 2.0 to 4.05
|
4.000 hours
Interval 2.0 to 6.03
|
4.017 hours
Interval 2.0 to 4.05
|
SECONDARY outcome
Timeframe: Day 1 (pre-dose, within 30 minutes of dosing), 0.5, 1, 2, 4, 6, 8 hours postdose (prior to Day 1 evening dose), 24 hours postdose (prior to Day 2 morning dose), 72 hours postdose (Day 4), on Day 7, and at the end of study (48 hours after last dose)Population: The PK population included all participants who received at least 1 dose of study drug and had at least 1 quantifiable post-dose blood sample collected for analysis of PTC923, BH4, Phe, or Tyr concentrations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=3 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=3 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Tmax of Phe and Tyr
Phe
|
0.250 hours
Interval 0.0 to 0.5
|
0.517 hours
Interval 0.0 to 8.17
|
0.000 hours
Interval 0.0 to 0.0
|
0.000 hours
Interval 0.0 to 0.0
|
|
Tmax of Phe and Tyr
Tyr
|
4.992 hours
Interval 3.87 to 7.37
|
4.025 hours
Interval 4.0 to 8.17
|
4.000 hours
Interval 4.0 to 7.03
|
2.000 hours
Interval 2.0 to 7.05
|
SECONDARY outcome
Timeframe: Baseline (Day 1, pre-dose); Day 7Population: The Efficacy population included all randomized participants who received any amount of study drug, and had available pre-dose Phe concentrations at Day 1 and at least 1 post-Day 1 visit within a given period. Here, 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=4 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=4 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Change From Baseline (Day 1) in Plasma Phe Concentration at Day 7
Baseline
|
884.20 μmol/L
Standard Deviation 975.507
|
675.68 μmol/L
Standard Deviation 842.579
|
1516.98 μmol/L
Standard Deviation 442.673
|
971.48 μmol/L
Standard Deviation 455.598
|
|
Change From Baseline (Day 1) in Plasma Phe Concentration at Day 7
Change at Day 7
|
-812.25 μmol/L
Standard Deviation 967.423
|
-622.02 μmol/L
Standard Deviation 840.853
|
-1428.19 μmol/L
Standard Deviation 531.327
|
-913.63 μmol/L
Standard Deviation 448.282
|
SECONDARY outcome
Timeframe: Day 7Population: The Efficacy population included all randomized participants who received any amount of study drug, and had available pre-dose Phe concentrations at Day 1 and at least 1 post-Day 1 visit within a given period.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=4 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=4 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Number of Participants With Phe Concentrations in Acceptable Treatment Range of 130 to 360 μmol/L at Day 7
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 7Population: The Efficacy population included all randomized participants who received any amount of study drug, and had available pre-dose Phe concentrations at Day 1 and at least 1 post-Day 1 visit within a given period.
Outcome measures
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 Participants
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 Participants
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=4 Participants
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=4 Participants
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Number of Participants With Normal Blood Phe Concentrations <130 μmol/L at Day 7
|
4 Participants
|
4 Participants
|
3 Participants
|
4 Participants
|
Adverse Events
Cohort 1: PTC923 2.5 mg/kg/Day
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 1: PTC923 10 mg/kg/Day
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 2: PTC923 5 mg/kg/Day
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Cohort 2: PTC923 20 mg/kg/Day
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1: PTC923 2.5 mg/kg/Day
n=4 participants at risk
Participants received PTC923 2.5 mg/kg/day for 7 days in Period 1.
|
Cohort 1: PTC923 10 mg/kg/Day
n=4 participants at risk
Participants received PTC923 10 mg/kg/day for 7 days in Period 2.
|
Cohort 2: PTC923 5 mg/kg/Day
n=4 participants at risk
Participants received PTC923 5 mg/kg/day for 7 days in Period 1.
|
Cohort 2: PTC923 20 mg/kg/Day
n=4 participants at risk
Participants received PTC923 20 mg/kg/day for 7 days in Period 2.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Vomiting
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Gastrointestinal disorders
Faeces discoloured
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Gastrointestinal disorders
Retching
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
General disorders
Fatigue
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
General disorders
Thirst
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Psychiatric disorders
Enuresis
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Psychiatric disorders
Irritability
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Psychiatric disorders
Restlessness
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Nervous system disorders
Psychomotor hyperactivity
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
50.0%
2/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Nervous system disorders
Headache
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Infections and infestations
Eyelid infection
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
|
Vascular disorders
Pallor
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
25.0%
1/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
0.00%
0/4 • From first dose of study drug (Day 1) up to 30 (±3) days after last dose of study drug (up to 50 days)
The Safety population included all randomized participants who received any amount of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER