NCT05025332

An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (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 Pitt Hopkins 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 Pitt Hopkins 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

• Pitt Hopkins 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: 2022-10-14
• Primary completion: 2024-02-27
• Completion: 2024-05-03
• First posted: 2021-08-27
• Last updated: 2025-06-18
• Results posted: 2025-05-18

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study