NCT07593391 An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
| NCT ID | NCT07593391 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Neuren Pharmaceuticals Limited |
| Condition | Phelan-McDermid Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 180 participants |
| Start Date | 2026-06-08 |
| Primary Completion | 2028-10-29 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 180 participants in total. It began in 2026-06-08 with a primary completion date of 2028-10-29.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This Phase 3, open-label extension, multicenter study will evaluate long-term safety, tolerability and efficacy of NNZ-2591 in pediatric participants with Phelan- McDermid Syndrome.
Eligibility Criteria
Inclusion Criteria: 1. 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. 2. Participant must have completed all applicable study visits for the antecedent study in which they participated. 3. Body weight ≥ 10 kg at Screening/Baseline. 4. Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits. 5. Not actively undergoing regression or loss of skills. Exclusion Criteria: 1. Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol. 2. Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications). 3. 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. 4. 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. 5. Abnormal liver function laboratory results during the Screening period, as defined by the protocol 6. Abnormal QT interval on Screening ECG as defined by the protocol.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07593391 clinical trial?
This trial is open to participants of all sexes, aged 3 Years or older, up to 12 Years, studying Phelan-McDermid Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07593391 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 180 participants.
Is NCT07593391 currently recruiting?
Yes, NCT07593391 is actively recruiting participants. Contact the research team at medicalinformation@neurenpharma.com for enrollment information.
Where is the NCT07593391 trial being conducted?
This trial is being conducted at Glendale, United States, San Rafael, United States, Chevy Chase, United States, Lexington, United States and 2 additional locations.
Who is sponsoring the NCT07593391 clinical trial?
NCT07593391 is sponsored by Neuren Pharmaceuticals Limited. The trial plans to enroll 180 participants.