Actively Recruiting
A Phase 3 Open-label Extension Study to Investigate the Long-term Safety and Efficacy of Orally Administered NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Led by Neuren Pharmaceuticals Limited · Updated on 2026-05-22
180
Participants Needed
1
Research Sites
2 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the long-term safety, tolerability, and effectiveness of NNZ-2591 in children with Phelan-McDermid Syndrome in this Phase 3, open-label extension, multicenter study. This study focuses on pediatric participants who have previously taken part in related NNZ-2591 studies. The aim is to better understand how this oral medication works over an extended period in this specific group. Participants who qualify will receive NNZ-2591 orally twice daily for 52 weeks during the Treatment Period. This is followed by a 2-week Safety Follow-Up to monitor any effects after treatment ends. The total time each participant spends in the study is up to 56 weeks, allowing researchers to observe the medication's impact over nearly a year. During the study, children will undergo various assessments including symptom severity checks and baseline characteristics before starting treatment. Researchers will monitor safety through adverse event tracking, physical exams, laboratory tests, and heart monitoring with ECGs. Effectiveness will be measured using several scales designed to assess changes in symptoms and behavior over months 3 and 12. Participants' health and responses will be closely followed throughout the study and safety follow-up period.
CONDITIONS
Brief Title
An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Who Can Participate
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 10 kg at Screening/Baseline.
- Participants with a PMSA-S overall score 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.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - 52 weeks
Participants receive the study drug NNZ-2591 orally twice daily during the treatment period.
Visits occur at baseline and months 3 and 12 for assessments
Duration - 2 weeks
Participants undergo a safety follow-up period after completing treatment to monitor for any adverse effects.
1 visit (in-person) for safety follow-up
Trial Site Locations
Total: 1 location
1
Neuren PMS-302 Site#111
San Rafael, California, United States, 94903
Actively Recruiting
Research Team
M
Medical Information Lead
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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