What is NNZ-2591 studying for Phelan-McDermid Syndrome?

NNZ-2591 is being studied to understand its long-term safety, tolerability, and effectiveness in children with Phelan-McDermid Syndrome. The research evaluates how this oral medication affects symptoms over a year. The study is led by Neuren Pharmaceuticals Limited and focuses on improving knowledge for this rare genetic condition.

Who may be eligible for this Phelan-McDermid Syndrome clinical trial?

Children aged 3 to 12 years diagnosed with Phelan-McDermid Syndrome who have participated in previous NNZ-2591 studies may be eligible. Participants must weigh at least 10 kg and not be currently losing skills or experiencing seizures beyond specific limits. Please review eligibility criteria for more details.

Is there any cost to participate in this Phelan-McDermid Syndrome clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants and families should plan accordingly.

Actively Recruiting

Phase 3

Age: 3Years - 12Years

All Genders

ID07593391

Long-term Study of Oral NNZ-2591 Treatment in Children Ages 3 to 12 With Phelan-McDermid Syndrome

Led by Neuren Pharmaceuticals Limited · Updated on 2026-08-12

180

Participants Needed

Research Sites

2 weeks

Total Duration

AI-Summary

What this Trial Is About

This research aims to evaluate the long-term safety, tolerability, and effectiveness of NNZ-2591 in children with Phelan-McDermid Syndrome. It is a Phase 3, open-label extension study involving pediatric participants who have previously taken part in related studies. The study focuses on assessing how NNZ-2591 affects symptom severity and overall health over an extended period. Participants will receive NNZ-2591 orally twice daily during a 52-week treatment period. Before starting treatment, eligibility will be confirmed through assessments including baseline characteristics and symptom severity. After completing the treatment phase, participants will enter a 2-week safety follow-up to monitor any ongoing effects or side effects. Throughout the study, participants will undergo various evaluations including clinical assessments, ECG monitoring, laboratory tests, and symptom severity scales. Safety and tolerability will be measured by tracking adverse events, vital signs, and laboratory results. Efficacy will be assessed using standardized tools such as the Phelan-McDermid Syndrome Assessment of Change and Vineland Adaptive Behavior Scales. Overall participation may last up to 56 weeks.

CONDITIONS

Brief Title

An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

Who Can Participate

Age: 3Years - 12Years

All Genders

Eligibility Criteria

You may qualify if you...

Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent for the antecedent study.
Participant must have completed all applicable study visits for the antecedent study in which they participated.
Body weight �3e= 10 kg at Screening/Baseline.
Participants with a PMSA-S overall score �3e= 3 at the Screening and Baseline visits.
Not actively undergoing regression or loss of skills.

You will not qualify if you...

Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol.
Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications).
Psychotropic medications or any other medication used for a chronic illness (not including antibiotics, pain relievers, anti-diarrheals, and laxatives) with doses and dosing regimen that have not been stable for at least 4 weeks before Screening. If the treatment was discontinued, the discontinuation must have occurred no fewer than 2 weeks before the start of Screening.
Any intercurrent seizures in the past 6 months and /or more than 1 seizure in the past 12 months.
A single febrile seizure in the 6 months prior to screening is allowable if no rescue medication was required.
Abnormal liver function laboratory results during the Screening period, as defined by the protocol
Abnormal QT interval on Screening ECG as defined by the protocol.

Research Team

Medical Information Lead

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here