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

Completed · Phase 2

Conditions studied: Pitt Hopkins Syndrome

In brief

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

Key facts

Study ID

NCT05025332

Run by

Neuren Pharmaceuticals Limited

People needed

28

Starts

2022-10-14

Expected to finish

2024-05-03

Last updated by the study team

2025-06-18

Who can join

Age: 3 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Body weight <12kg at screening
• Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
• Abnormal QTcF interval or prolongation at Screening.
• Any other clinically significant finding on ECG at the Screening visit.
• Positive for severe acute respiratory syndrome coronavirus 2 (SARSCoV-2) and previous COVID 19 infection with last 12 months that required hospitalization.
• Unstable or changes Psychotropic treatment 2 weeks prior to screening
• Excluded concomitant treatments.
• Actively undergoing regression or loss of skills.
• Unstable seizure profile.
• Current clinically significant renal conditions and abnormalities
• Current clinically significant cardiovascular, hepatic, gastrointestinal, respiratory, endocrine disease, or clinically significant organ impairment.
• Current clinically significant hypo- or hyperthyroidism, Type 1 or Type 2 diabetes mellitus requiring insulin (whether well controlled or uncontrolled), or uncontrolled Type 1 or Type 2 diabetes.
• Has planned surgery during the study.
• History of, or current, cerebrovascular disease or brain trauma.
• History of, or current catatonia or catatonia-like symptoms.
• History of, or current, malignancy.
• Current major or persistent depressive disorder (including bipolar depression).
• Significant, uncorrected visual or uncorrected hearing impairment.
• Allergy to strawberry.
• Positive pregnancy test
• Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• University of California at San Francisco — San Francisco, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Rush University Medical Center — Chicago, Illinois, United States
• UT Southwestern — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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