An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
NCT ID: NCT05879614
Last Updated: 2024-12-09
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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WITHDRAWN
PHASE2
INTERVENTIONAL
2023-09-01
2025-06-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Interventions
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NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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.
4 Years
12 Years
ALL
No
Sponsors
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Neuren Pharmaceuticals Limited
INDUSTRY
Responsible Party
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Principal Investigators
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Jordan Press
Role: STUDY_DIRECTOR
Neuren Pharmaceuticals
Locations
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Rady Children's Hospital San Diego
San Diego, California, United States
Rare Disease Research
Atlanta, Georgia, United States
Uncommon Cures
Chevy Chase, Maryland, United States
Suburban Research
Media, Pennsylvania, United States
Countries
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Other Identifiers
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NEU-2591-PWS-001
Identifier Type: -
Identifier Source: org_study_id