NCT05025241

An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Neuren Pharmaceuticals Limited · Industry
• Sex: All
• Age: 3 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 Phelan-McDermid 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 Phelan-McDermid 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

• Phelan-McDermid Syndrome

Interventions

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

Timeline

• Start date: 2022-08-08
• Primary completion: 2023-11-01
• Completion: 2023-11-17
• First posted: 2021-08-27
• Last updated: 2025-06-03
• Results posted: 2025-05-30

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study