NCT05011851

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Neuren Pharmaceuticals Limited · Industry
• Sex: All
• Age: 3 Years – 17 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 Angelman syndrome

Detailed description

The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution, 50mg/L, in children and adolescents with Angelman syndrome. The secondary purpose is to investigate measures of efficacy of subjects will receive treatment of 50mg/mL orally administered NNZ-2591 for a total of 13 weeks

Conditions

• Angelman 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: 2022-07-12
• Primary completion: 2024-05-13
• Completion: 2024-07-16
• First posted: 2021-08-18
• Last updated: 2025-01-31

Locations

3 sites across 1 country: Australia

Regulatory

• FDA-regulated drug study