NCT07281079 A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
| NCT ID | NCT07281079 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Neuren Pharmaceuticals Limited |
| Condition | Phelan-McDermid Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 160 participants |
| Start Date | 2025-11-12 |
| Primary Completion | 2027-10-31 |
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 160 participants in total. It began in 2025-11-12 with a primary completion date of 2027-10-31.
⚠ 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, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study will evaluate the efficacy and safety of NNZ-2591 compared to placebo 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. 2. Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK3. 3. Body weight ≥ 10 kg at Screening. 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. Current treatment with more than 3 allowable psychotropic medications. 3. Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications). 4. 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. 5. 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. 6. Abnormal liver function laboratory results during the Screening period, as defined by the protocol 7. Abnormal QT interval on Screening ECG as defined by the protocol.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07281079 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 NCT07281079 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 160 participants.
Is NCT07281079 currently recruiting?
Yes, NCT07281079 is actively recruiting participants. Contact the research team at medicalinformation@neurenpharma.com for enrollment information.
Where is the NCT07281079 trial being conducted?
This trial is being conducted at Birmingham, United States, Glendale, United States, Palo Alto, United States, San Diego, United States and 11 additional locations.
Who is sponsoring the NCT07281079 clinical trial?
NCT07281079 is sponsored by Neuren Pharmaceuticals Limited. The trial plans to enroll 160 participants.