Trial Outcomes & Findings for An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001) (NCT NCT05025241)
NCT ID: NCT05025241
Last Updated: 2025-06-03
Results Overview
To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.
COMPLETED
PHASE2
18 participants
13 weeks
2025-06-03
Participant Flow
Participants were recruited based on physician referral at 4 academic medical centers between August 2022 and June 2023. The first participant entered screening on 08 August 2022, and the last participant entered screening on 22 June 2023. Of 23 consented participants, 18 met eligibility criteria and were enrolled into the study. Enrolled participants received NNZ2591 12 mg/kg by liquid oral dose twice daily
Following consent, participants underwent 4 weeks of screening for eligibility. If successful, participants were enrolled and commenced open-label active treatment.
Participant milestones
| Measure |
NNZ-2591
All enrolled participants
|
|---|---|
|
Screening
STARTED
|
23
|
|
Screening
COMPLETED
|
18
|
|
Screening
NOT COMPLETED
|
5
|
|
Treatment and Follow Up
STARTED
|
18
|
|
Treatment and Follow Up
COMPLETED
|
15
|
|
Treatment and Follow Up
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
NNZ-2591
All enrolled participants
|
|---|---|
|
Screening
Failed Screening
|
5
|
|
Treatment and Follow Up
Adverse Event
|
3
|
Baseline Characteristics
Only 2 participants recorded seizures at enrollment
Baseline characteristics by cohort
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
Age, Continuous
|
8.6 years
STANDARD_DEVIATION 2.7 • n=18 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=18 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race : White
|
16 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race : Black or African American
|
1 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race : Asian
|
0 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race : Native Hawaiian or other Pacific Islander
|
0 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race : American Indian or Alaska Native
|
0 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race : Multi Racial
|
1 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race : Other
|
0 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Ethnicity : Hispanic or Latino
|
3 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Ethnicity : Not Hispanic or Latino
|
15 Participants
n=18 Participants
|
|
Height (cm)
|
130.6 cm
STANDARD_DEVIATION 15.15 • n=18 Participants
|
|
Weight (kg)
|
30.4 kg
STANDARD_DEVIATION 10.75 • n=18 Participants
|
|
BMI (kg/m^2)
|
17.4 kg/m^2
STANDARD_DEVIATION 3.48 • n=18 Participants
|
|
PMS Genotype
Terminal Deletion - Class I
|
7 Participants
n=18 Participants
|
|
PMS Genotype
Terminal Deletion - Class II
|
3 Participants
n=18 Participants
|
|
PMS Genotype
Ring 22
|
0 Participants
n=18 Participants
|
|
PMS Genotype
Unbalanced Translocation
|
0 Participants
n=18 Participants
|
|
PMS Genotype
Interstitial Deletion
|
1 Participants
n=18 Participants
|
|
PMS Genotype
SHANK 3 variant/mutation
|
6 Participants
n=18 Participants
|
|
PMS Genotype
Other
|
1 Participants
n=18 Participants
|
|
History of regression
Yes
|
11 Participants
n=18 Participants
|
|
History of regression
No
|
7 Participants
n=18 Participants
|
|
Autism Mental Status Exam
|
7.8 score on a scale
STANDARD_DEVIATION 2.05 • n=18 Participants
|
|
Seizure Types
Simple Partial
|
0 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Complex Partial
|
1 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Atonic
|
1 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Tonic
|
1 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Clonic
|
0 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Tonic-Clonic
|
1 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Myoclonic
|
0 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Absence/Atypical Absence
|
0 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Not Actual Seizure/Spells
|
0 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Types
Other
|
1 Participants
n=2 Participants • Only 2 participants recorded seizures at enrollment
|
|
Seizure Characteristics at baseline
Change in awareness
|
2 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Loss of urine or bowel control
|
0 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Loss of ability to communicate
|
2 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Automatic repeated movements
|
0 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Rhythmic jerking muscle
|
2 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Falls if sitting or standing
|
0 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Head drops
|
1 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Warning before seizure occurred
|
0 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Common triggers
|
0 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Muscle stiffness
|
1 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Muscle twitch
|
1 participants
n=18 Participants
|
|
Seizure Characteristics at baseline
Other
|
1 participants
n=18 Participants
|
|
Stanford-Binet Intelligence Scale (NVIQ) Standard Score
|
47.7 score on a scale
STANDARD_DEVIATION 5.39 • n=9 Participants • Participants who could not be assessed on the Stanford-Binet Intelligence (SB-5) due to their developmental level were assessed using the Mullen Scales of Early Learning instead.
|
|
Stanford-Binet Intelligence Scale (NVIQ) z deviation score
|
40.9 score on a scale
STANDARD_DEVIATION 17.2 • n=9 Participants • Participants who could not be assessed on the Stanford-Binet Intelligence (SB-5) due to their developmental level were assessed using the Mullen Scales of Early Learning instead.
|
|
Mullen Scales of Early Learning Non-Verbal Developmental Quotient Score
|
15.7 score on a scale
STANDARD_DEVIATION 7.25 • n=8 Participants • Includes only participants who were unable to be assessed on the Stanford-Binet Intelligence (SB-5) due to their developmental level.
|
|
Presence of co-morbid psychiatric disorders
Autism Spectrum Disorder (ASD)
|
14 participants
n=18 Participants
|
|
Presence of co-morbid psychiatric disorders
BiPolar Disorders
|
0 participants
n=18 Participants
|
|
Presence of co-morbid psychiatric disorders
Attention Deficit Disorder
|
5 participants
n=18 Participants
|
|
Presence of co-morbid psychiatric disorders
Obsessive Compulsive Disorder
|
0 participants
n=18 Participants
|
|
Presence of co-morbid psychiatric disorders
Other
|
1 participants
n=18 Participants
|
PRIMARY outcome
Timeframe: 13 weeksPopulation: Intention to treat population
To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
Safety and Tolerability
Participants with any AE
|
17 Participants
|
|
Safety and Tolerability
Participants with TEAE
|
17 Participants
|
|
Safety and Tolerability
Participants with Serious TEAE
|
1 Participants
|
|
Safety and Tolerability
Participants who discontinued due to TEAE
|
3 Participants
|
|
Safety and Tolerability
Participants with mild TEAE
|
11 Participants
|
|
Safety and Tolerability
Participants with moderate TEAE
|
5 Participants
|
|
Safety and Tolerability
Participants with severe TEAE
|
1 Participants
|
PRIMARY outcome
Timeframe: Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13.Population: PK Population
Approximately nine sparse pharmacokinetic (PK) samples were collected from each participant under steady-state conditions. These samples were taken at pre-dose, 1-3 hours post-dose, and/or 4-7 hours post-dose during Weeks 2, 6, and 13. The individual pharmacokinetic parameters for NNZ-2591, including half-life (t1/2) and area under the curve over 24 hours (AUC24), were derived using subject-level concentration-time profiles from the study population model.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
Pharmacokinetic - Mean AUC24
|
411 µg.h/mL
Standard Deviation 116
|
PRIMARY outcome
Timeframe: Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13.Population: All participants enrolled in this study who receive the study drug through to the morning dose of Week 2 (Visit 5) as a minimum, and who undergo PK sample collection at least one of the specified post-dose time point(s).
Approximately nine sparse pharmacokinetic (PK) samples were collected from each participant under steady-state conditions. These samples were taken at pre-dose, 1-3 hours post-dose, and/or 4-7 hours post-dose during Weeks 2, 6, and 13. The individual pharmacokinetic parameters for NNZ-2591, including half-life (t1/2) and area under the curve over 24 hours (AUC24), were derived using subject-level concentration-time profiles from the study population model.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
Pharmacokinetic - t1/2
|
8.02 hours
Standard Deviation 2.33
|
SECONDARY outcome
Timeframe: CGI-I was assessed at Weeks 6, 13/EOT & 15. Overall improvement scores relate to Week 13/EOT visit.Population: Intention to treat
Phelan-McDermid Syndrome-specific Clinical Global Impression of Improvement Scale (CGI-I) - Overall Improvement Score on a 7 point Likert scale (1-7) where lower scores are better.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
CGI-I
|
2.4 score on a scale
Standard Deviation 0.86
|
SECONDARY outcome
Timeframe: CIC was assessed at Week13/EOTPopulation: Intention to treat population
Caregiver Impression of Improvement: Measured on a 7 point Likert scale (1-7) where lower scores are better.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
CIC
|
2.7 score on a scale
Standard Deviation 1.02
|
SECONDARY outcome
Timeframe: Change in score assessed from baseline (visit 3, week 0) to visit 16 (week 13/EOT).Population: Intention to treat
Phelan-McDermid syndrome-specific Clinical Global Impression Scale-Severity (CGI-S) -Change from baseline on Overall Score. Based on a 7 point Likert scale (1-7) where a lower score is better.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
CGI-S
|
-0.4 score on a scale
Standard Deviation 0.5
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall improvement score.Population: Intention to treat population
Caregiver Top 3 Concerns - Total Concerns Severity: Change from baseline. The range of scores was (0-30) for total concerns, with higher scores being worse
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
Top 3 Concerns
|
-5.9 score on a scale
Standard Deviation 5.86
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16).Population: Intention to treat population
MacArthur-Bates Communicative Development Inventory (MB-CDI) - change from baseline. Range of scores was (0-792) with higher scores being better.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
MB-CDI
|
12.3 score on a scale
Standard Deviation 35.19
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in Total Score.Population: Intention to treat population
Observer-Reported Communication Ability (ORCA) - Change from baseline in Total Score. Range of Scores was (25.8-83.8) with higher scores being better
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
ORCA
|
1.9 score on a scale
Standard Deviation 4.22
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total score.Population: Intention to treat population
Aberrant Behavior Checklist-2 (ABC-2) - Total score: Change from baseline. Range of scores was (0-174) with higher scores being worse
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
ABC-2
|
-17.2 score on a scale
Standard Deviation 19.65
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total score.Population: Intention to treat population
Child Sleep Habits Questionnaire (CSHQ) - Change from baseline in Total Score. Range of scores was (33-99) with higher scores being worse.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
CSHQ
|
-3.6 score on a scale
Standard Deviation 5.68
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total frequency score.Population: Intention to treat population
Gastrointestinal Health Questionnaire (GIHQ) - Total Frequency Score: Change from Baseline. Range of scores was (0-212) with higher scores being worse.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
GIHQ
|
-9.6 score on a scale
Standard Deviation 10.8
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in composite standard score.Population: Intention to treat population
Vineland Adaptive Behavior Scales-3, Change from baseline in Composite Standard Score. Range of scores was (20-140) with higher scores being better.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
VABS-3
|
2.8 score on a scale
Standard Deviation 7.82
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall score score.Population: Intention to treat population
Quality of Life Inventory-Disability (QL-Disability) Overall Score - change from baseline. Range of scores was (0-100) with higher scores being better.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
QL-Disability
|
6.1 score on a scale
Standard Deviation 8.91
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall quality of life rating score.Population: Intention to treat population
Impact of Childhood Neurological Disability (ICND) - Change from baseline in overall quality of life rating. Range of (1- 6) for quality of life rating, with higher scores indicating greater impact.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
ICND
|
0.3 score on a scale
Standard Deviation 0.69
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall severity score.Population: Intention to treat population
PMS Clinician Domain Specific Rating Scale - Change from baseline in overall severity score. Range of Scores Was (0-20) With Higher Scores Being Worse.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
PMS-DSRS
|
-0.9 score on a scale
Standard Deviation 1.69
|
SECONDARY outcome
Timeframe: Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total frequency score.Population: Intention to treat population
Total Frequency Score - Change from Baseline. Range of scores was (0-4) for each of 30 behaviors, total range (0-120), with lower scores being better.
Outcome measures
| Measure |
NNZ-2591
n=18 Participants
All enrolled participants
|
|---|---|
|
Behavior Problems Inventory - Short Form
|
-5.1 score on a scale
Standard Deviation 9.41
|
Adverse Events
NNZ-2591
Serious adverse events
| Measure |
NNZ-2591
n=18 participants at risk
All enrolled participants
|
|---|---|
|
Gastrointestinal disorders
Gastroenteritis
|
5.6%
1/18 • Number of events 1 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
Other adverse events
| Measure |
NNZ-2591
n=18 participants at risk
All enrolled participants
|
|---|---|
|
Gastrointestinal disorders
Constipation
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Gastrointestinal disorders
Diarrhea
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Gastrointestinal disorders
Nausea
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
General disorders
Fatigue
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
General disorders
Pyrexia
|
16.7%
3/18 • Number of events 3 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Infections and infestations
Covid 19
|
16.7%
3/18 • Number of events 3 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Infections and infestations
Nasopharyngitis
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Infections and infestations
Otitis Media
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
16.7%
3/18 • Number of events 3 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Nervous system disorders
Psychomotor Hyperactivity
|
22.2%
4/18 • Number of events 4 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Nervous system disorders
Somnolence
|
16.7%
3/18 • Number of events 3 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Psychiatric disorders
Aggression
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Psychiatric disorders
Insomnia
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
11.1%
2/18 • Number of events 2 • 15 weeks for each participant starting on date of first dose
Once eligibility was confirmed, subjects were dosed at a starting dose of 4 mg/kg twice daily (BID). Doses were titrated to 8 mg/kg at week 2 and to 12 mg/kg at week 6. The mg/kg dosing was based on the subject's weight at baseline. Subjects received treatment with NNZ-2591 for a total of 13 weeks.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60