Trial · Interventional
WithdrawnPhase 2Registry dataAn Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
Short name PWS-001
This trial was withdrawn before it enrolled anyone.
Reason given at the registry: Revised development strategy
Overview
- Status
- Withdrawn
- Phase
- Phase 2
- Enrollment
- 0 participants (actual)
- Sponsor
- Neuren Pharmaceuticals Limited · Industry
- Design
- Allocation not applicable · Open label (no masking)
- Countries
1 country
United States
- Start
- 1 Sept 2023
- Primary completion
- 30 Jun 2025 (estimated)
- Completion
- 30 Jun 2025 (estimated)
- First posted
- 30 May 2023
- Last update posted
- 9 Dec 2024
- Conditions
- Prader-Willi Syndrome
Timeline
1 trial · 1 stopped early
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome.
Interventions
- NNZ-2591Drug
Arms1
Experimental
NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Primary outcomes5
Outcomes the sponsor listed as primary.
Safety and Tolerability
Time frame · 13 weeks
Registry description
To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.
Pharmacokinetic - Measurement of Cmax
Time frame · 13 weeks
Registry description
Maximum observed concentration (Cmax) of NNZ-2591
Pharmacokinetic - Measurement of AUC
Time frame · 13 weeks
Registry description
Area under the concentration-time curve of NNZ-2591
Pharmacokinetic - Measurement of time to Cmax
Time frame · 13 weeks
Registry description
Time to Cmax of NNZ-2591
Pharmacokinetic - Measurement of t1/2
Time frame · 13 weeks
Registry description
Apparent terminal elimination half-life of NNZ-2591
Secondary outcomes(17)
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by PWS-specific Clinical Global Impression Scale \& Domain -Overall Improvement Score (CGI-I)
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Caregiver Global Impression-Change Score
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by PWS-specific Clinical Global Impression Scale-Severity (CGI-S) Overall Score
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Caregiver Top 3 Concerns Likert Scale Scores
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by PWS Profile Score
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by PWS Anxiousness and Distress Behaviors Questionnaire Score (PADQ)
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Autism Diagnostic Observation Schedule (ADOS-2), Repetitive behaviors and Social scores
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Hyperphagia Questionnaire-Clinical Trials (HQ-CT) Score
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Food Safety Zone Questionnaire Score
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Vineland Adaptive Behavior Scales-3 Growth Scale Scores
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Zarit Burden Interview Score
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Child Sleep Habits Questionnaire (CSHQ)
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Impact of Childhood Neurological Disability Scale (ICND)
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Quality of Life Inventory-Disability (QI-Disability)
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Kaufman Brief Intelligence Test or Mullen Scales of Early Learning
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by PWS Suicidality Assessment
Exploratory efficacy measurement
Time frame · 13 weeks
Registry description
Assessed by Caregiver Diary
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
No results are posted for this trial.
A trial that is still running, or that stopped early, may post results later.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 4 Years up to 12 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
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