NCT05879614

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

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Neuren Pharmaceuticals Limited · Industry
• Sex: All
• Age: 4 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution in children and adolescents with Prader-Willi Syndrome. The secondary purpose is to investigate measures of efficacy. Subjects will receive treatment with NNZ-2591 oral solution (50 mg/mL) doses for a total of 13 weeks.

Conditions

• Prader-Willi Syndrome

Interventions

Type	Name	Description
DRUG	NNZ-2591	NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.

Timeline

• Start date: 2023-09-01
• Primary completion: 2025-06-30
• Completion: 2025-06-30
• First posted: 2023-05-30
• Last updated: 2024-12-09

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study