Clinical trials: SHANK3 Haploinsufficiency
Phelan-McDermid Syndrome, SHANK3 Haploinsufficiency
RB001 Gene Therapy Study in Children With SHANK3-related Phelan-McDermid Syndrome (PMS)
This is an open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of RB001 administered via intracerebroventricular (ICV) injection in pediatric with SHANK3 related…
- Trial name
- NCT07014020
- Testing
- RB001
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking University First Hospital
Autism Spectrum Disorder
Effect of Lithium in Patients With Autism Spectrum Disorder and Phelan-McDermid Syndrome (SHANK3 Haploinsufficiency)
There is currently no treatment for the body symptoms of Autism Spectrum Disorders (ASD). However, basic research suggests that some forms of ASD may be alleviated, even in the adult stage.
- Trial name
- Lisphem (NCT04623398)
- Testing
- Lithium Carbonate
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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