Clinical trials: SHANK3 Haploinsufficiency, open to volunteers
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
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)
This is a long-term follow-up study of participants in the RB001-101 gene replacement therapy clinical trial for Children With SHANK3-related Phelan-McDermid Syndrome (PMS).
- Trial name
- RB001-101-LTFU (NCT07690527)
- Testing
- RB001
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking University First Hospital