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

Who is this study for? Patients with Pitt Hopkins Syndrome
What treatments are being studied? NNZ-2591
Status: Not yet recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 17
Healthy Volunteers: No
View:

• Clinical diagnosis of PTHS with a documented disease-causing genetic etiology for the disorder.

• Males or females aged 3-17 years.

• Body weight of 12kg or higher at screening

• Subjects with a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at the Screening visit.

• Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit

• Each subject must be able to swallow the study medication provided as a liquid solution.

• Caregiver(s) must have sufficient English language skills.

Locations
United States
Alabama
University of Alabama at Birmingham
Birmingham
California
University of California at San Francisco
San Francisco
Colorado
Children's Hospital Colorado
Aurora
Texas
UT Southwestern
Dallas
Time Frame
Start Date: April 28, 2022
Estimated Completion Date: May 18, 2023
Participants
Target number of participants: 20
Treatments
Experimental: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.
Sponsors
Leads: Neuren Pharmaceuticals Limited

This content was sourced from clinicaltrials.gov

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