Clinical trials: 22q13 Deletion Syndrome
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
JAG201 Gene Therapy Study in Children & Adults 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 administered via intracerebroventricular (ICV) injection in pediatric…
- Trial name
- NCT06662188
- Testing
- JAG201
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Jaguar Gene Therapy, LLC
Phelan-McDermid Syndrome
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.
- Trial name
- PMS-001 (NCT05025241)
- Testing
- NNZ-2591
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Neuren Pharmaceuticals Limited
Phelan-McDermid Syndrome
An Open-label Study of 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 Phelan- McDermid Syndrome.
- Trial name
- NCT07593391
- Testing
- NNZ-2591
- Where
- 11 sites in United States
- Volunteers
- 180 people
- Sponsor
- Neuren Pharmaceuticals Limited
Phelan-McDermid Syndrome
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
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.
- Trial name
- NCT07281079
- Testing
- NNZ-2591
- Where
- 18 sites in United States, Canada
- Volunteers
- 160 people
- Sponsor
- Neuren Pharmaceuticals Limited