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Clinical Trial Summary

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


Clinical Trial 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) at weight-banded doses for a total of 13 weeks. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05025332
Study type Interventional
Source Neuren Pharmaceuticals Limited
Contact James Shaw
Phone +61 427 299 669
Email [email protected]
Status Not yet recruiting
Phase Phase 2
Start date October 1, 2021
Completion date September 30, 2022

See also
  Status Clinical Trial Phase
Recruiting NCT04132427 - MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders Phase 2
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford