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Trial · Interventional

WithdrawnPhase 2Registry data

An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)

Short name PWS-001

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Withdrawn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
2023202420252026
  1. Ph 2

    PWS-001

    NCT05879614

    1 Sept 2023 to est. 30 Jun 2025

    Withdrawn

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.

  1. 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.

  2. Pharmacokinetic - Measurement of Cmax

    Time frame · 13 weeks

    Registry description

    Maximum observed concentration (Cmax) of NNZ-2591

  3. Pharmacokinetic - Measurement of AUC

    Time frame · 13 weeks

    Registry description

    Area under the concentration-time curve of NNZ-2591

  4. Pharmacokinetic - Measurement of time to Cmax

    Time frame · 13 weeks

    Registry description

    Time to Cmax of NNZ-2591

  5. Pharmacokinetic - Measurement of t1/2

    Time frame · 13 weeks

    Registry description

    Apparent terminal elimination half-life of NNZ-2591

Secondary outcomes(17)
  1. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by PWS-specific Clinical Global Impression Scale \& Domain -Overall Improvement Score (CGI-I)

  2. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Caregiver Global Impression-Change Score

  3. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by PWS-specific Clinical Global Impression Scale-Severity (CGI-S) Overall Score

  4. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Caregiver Top 3 Concerns Likert Scale Scores

  5. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by PWS Profile Score

  6. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by PWS Anxiousness and Distress Behaviors Questionnaire Score (PADQ)

  7. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Autism Diagnostic Observation Schedule (ADOS-2), Repetitive behaviors and Social scores

  8. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Hyperphagia Questionnaire-Clinical Trials (HQ-CT) Score

  9. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Food Safety Zone Questionnaire Score

  10. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Vineland Adaptive Behavior Scales-3 Growth Scale Scores

  11. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Zarit Burden Interview Score

  12. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Child Sleep Habits Questionnaire (CSHQ)

  13. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Impact of Childhood Neurological Disability Scale (ICND)

  14. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Quality of Life Inventory-Disability (QI-Disability)

  15. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by Kaufman Brief Intelligence Test or Mullen Scales of Early Learning

  16. Exploratory efficacy measurement

    Time frame · 13 weeks

    Registry description

    Assessed by PWS Suicidality Assessment

  17. 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
Inclusion Criteria: 1. Clinical diagnosis of PWS with a documented disease-causing genetic abnormality of the chromosome 15q11-q13 confirmed by DNA methylation and microarray. 2. Males or females aged 4-12 years, inclusive. 3. Body weight of 12 kg to 100kg (inclusive) at Baseline. 4. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit. 5. Must currently be on treatment with growth hormone. 6. Each subject must be able to swallow the study medication provided as a liquid solution. 7. Caregiver(s) must have sufficient English language skills. 8. Subject and caregiver must reside in the US and have been resident in the US for at least 3 months prior to screening. Exclusion Criteria: 1. Body weight \<12 kg or \>100 kg at Baseline. 2. HbA1c values above 7% at the Screening visit. 3. Clinically significant abnormalities in safety laboratory tests and vital signs at Screening. 4. Positive pregnancy test at the Screening visit. 5. Positive drugs of abuse screen not explained by concomitant medications. 6. Abnormal QTcF interval or prolongation at Screening. 7. Any other clinically significant finding on ECG at the Screening visit. 8. Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or Baseline. 9. Previous COVID 19 infection with last 12 months that required hospitalization. 10. Previous COVD-19 infection involving multi-organ systems, resulting in Multisystem Inflammatory Syndrome in Children (MIS-C) or with clinically significant long term effects. 11. COVID-19 infection associated with acute kidney injury (AKI) or renal conditions. 12. Renal conditions or abnormalities identified in laboratory testing, imaging or medical history. 13. Liver conditions and Hepatic abnormalities. 14. Vision abnormalities and Ocular conditions. 15. Excluded concomitant treatments. 16. Unstable seizure profile. 17. Current clinically significant cardiovascular, gastrointestinal, or respiratory disease, or clinically significant organ impairment, or endocrine disease with the exception of obesity and controlled hypothyroidism. 18. Current clinically significant hypo or hyperthyroidism, Type 1 or Type 2 diabetes mellitus requiring insulin (whether well controlled or uncontrolled), or uncontrolled Type 1 or Type 2 diabetes. 19. Has planned surgery during the study. 20. History of, or current, cerebrovascular disease or brain trauma. 21. History of, or current catatonia or catatonia-like symptoms. 22. History of, or current, malignancy. 23. Current major or persistent depressive disorder (including bipolar depression). 24. Significant uncorrected hearing impairment. 25. Allergy to strawberry. 26. Has participated in another interventional clinical study within 30 days prior to start of Screening. 27. Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study.

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