An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
About the study
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- 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.
EXCLUSION CRITERIA
Exclusion Criteria:
- 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.
- Has participated in another interventional clinical study within 30 days prior to start of Screening.
- Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study Details
Contition
Prader-Willi Syndrome
Age (in years)
4 - 12
Phase
PHASE2
Participants Needed
0
Est. Completion Date
Jun 30, 2025
Treatment Type
INTERVENTIONAL
Sponsor
Neuren Pharmaceuticals Limited
ClinicalTrials.gov NCT Identifier
NCT05879614
Study Number
NEU-2591-PWS-001
Understanding Clinical Trials
Get answers to your questions about clinical trials.What is clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?