SHANK3 Haploinsufficiency

Clinical trial pipeline · Data from ClinicalTrials.gov

See which SHANK3 Haploinsufficiency trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

RecruitingPhase 1/2

NCT06662188

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 ad…

United States6 participantsStarted 2024-01-07

Treatment: JAG201, JAG201

RecruitingNot Applicable

NCT07014020

RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

This is a first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of RB001 administered v…

China8 participantsStarted 2025-06-16

Treatment: RB001

All recent trials (3 shown)

CompletedPhase 3

NCT04623398

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 allevia…

France22 participantsStarted 2022-02-21

Treatment: Lithium Carbonate, Placebo