Neuren Pharmaceuticals Limited
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.
After providing informed consent/assent, pediatric participants with Phelan-McDermid syndrome who participated in previous studies (NEU-2591-PMS-301 and NEU-2591-PMS-001) will undergo assessments for eligibility, baseline characteristics and symptom severity. Once eligibility is confirmed, participants will receive orally administered NNZ-2591 during the 52-week Treatment Period. A 2-week safety follow-up period will occur immediately after the completion of the Treatment Period.
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 c…
The study drug will be administered twice daily orally.