This Phase 3, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study will evaluate the efficacy and safety of NNZ-2591 compare…

Phelan-McDermid Syndrome

~Estimated

~$10 – $8,750

Neuren Pharmaceuticals LimitedNCT07281079

Phase 3

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

This Phase 3, open-label extension, multicenter study will evaluate long-term safety, tolerability and efficacy of NNZ-2591 in pediatric participants with Phel…

Phelan-McDermid Syndrome

~Estimated

~$10 – $8,750

Neuren Pharmaceuticals LimitedNCT07593391

Phase 1

A Phase 1/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants With SHANK3 Haploinsufficiency

This is a Phase 1/2, first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of JAG201 a…

SHANK3 HaploinsufficiencyPhelan-McDermid Syndrome

~Estimated

~$10 – $8,750

Jaguar Gene Therapy, LLCNCT06662188