CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 28 enrolled
Drug / intervention
NNZ-2591drug
Likely dose
NNZ-2591 50mgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT05025332
NCT05025332Phase 2Completed

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

Neuren Pharmaceuticals Limited·interventional·Posted Aug 27, 2021·Updated Jun 18, 2025

In Brief

A Phase 2 clinical trial evaluating NNZ-2591 for Pitt Hopkins Syndrome. Completed, enrolled 28 participants across 5 sites.

Detailed Summary

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

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
20222023202420252026
First PostedAug 27, 2021
Enrollment StartOct 14, 2022
Primary CompletionFeb 27, 2024
Study CompletionMay 3, 2024
TodayJul 2, 2026
Enrollment to primary: 1.4 yearsPosted 4.8 years ago

Interventions

NNZ-2591drug

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