An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Start Date
6/8/2026
Completion Date
11/12/2028
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.
Detailed Description
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.
Eligibility Criteria
Age Range: 3 years to 12 years
Interventions
NNZ-2591
Conditions
Locations
Neuren PMS-302 Site #116
Birmingham, Alabama 35233
United States
Neuren PMS-302 Site#122
Glendale, California 91203
United States
Neuren PMS-302 Site#111
San Rafael, California 94903
United States
Neuren PMS-302 Site#109
Chevy Chase, Maryland 20815
United States
Neuren PMS-302 Site#106
Brookline, Massachusetts 02445
United States
Neuren PMS-302 Site#104
Lexington, Massachusetts 02421
United States
Neuren PMS-302 Site#101
New York, New York 10029
United States
Neuren PMS-302 Site#108
Cincinnati, Ohio 02421
United States
Neuren PMS-302 Site#115
Houston, Texas 77030
United States