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

Withdrawn before enrolling · Phase 2

Conditions studied: Prader-Willi Syndrome

In brief

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome.

Key facts

Study ID

NCT05879614

Run by

Neuren Pharmaceuticals Limited

People needed

0

Starts

2023-09-01

Expected to finish

2025-06-30

Last updated by the study team

2024-12-09

Who can join

Age: 4 and older, up to 12. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Clinical diagnosis of PWS with a documented disease-causing genetic abnormality of the chromosome 15q11-q13 confirmed by DNA methylation and microarray.
• Males or females aged 4-12 years, inclusive.
• Body weight of 12 kg to 100kg (inclusive) at Baseline.
• Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit.
• Must currently be on treatment with growth hormone.
• Each subject must be able to swallow the study medication provided as a liquid solution.
• Caregiver(s) must have sufficient English language skills.
• Subject and caregiver must reside in the US and have been resident in the US for at least 3 months prior to screening.

You may not qualify if…

• Body weight <12 kg or >100 kg at Baseline.
• HbA1c values above 7% at the Screening visit.
• Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
• Positive pregnancy test at the Screening visit.
• Positive drugs of abuse screen not explained by concomitant medications.
• Abnormal QTcF interval or prolongation at Screening.
• Any other clinically significant finding on ECG at the Screening visit.
• Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or Baseline.
• Previous COVID 19 infection with last 12 months that required hospitalization.
• Previous COVD-19 infection involving multi-organ systems, resulting in Multisystem Inflammatory Syndrome in Children (MIS-C) or with clinically significant long term effects.
• COVID-19 infection associated with acute kidney injury (AKI) or renal conditions.
• Renal conditions or abnormalities identified in laboratory testing, imaging or medical history.
• Liver conditions and Hepatic abnormalities.
• Vision abnormalities and Ocular conditions.
• Excluded concomitant treatments.
• Unstable seizure profile.
• Current clinically significant cardiovascular, gastrointestinal, or respiratory disease, or clinically significant organ impairment, or endocrine disease with the exception of obesity and controlled hypothyroidism.
• 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.
• Has planned surgery during the study.
• History of, or current, cerebrovascular disease or brain trauma.
• History of, or current catatonia or catatonia-like symptoms.
• History of, or current, malignancy.
• Current major or persistent depressive disorder (including bipolar depression).
• Significant uncorrected hearing impairment.
• Allergy to strawberry.

Where it is running

• 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

Full record on ClinicalTrials.gov

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