Gene therapy for children with SHANK3-related condition
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a gene therapy called JAG201 for children with a SHANK3 mutation or 22q13.3 deletion, which causes Phelan-McDermid syndrome. The goal is to see if it can help with development and daily function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- A child between 2 and 9 years old at the time of treatment
- Genetic test confirmed a loss-of-function mutation in SHANK3 or a certain type of 22q13.3 deletion
- Significant developmental or cognitive delay (IQ or score 70 or below)
- Commit to ongoing therapies like physical, speech, or behavioral therapy during the study
- Be a permanent legal resident of the continental U.S.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a new medicine, NNZ-2591, to see if it helps children with Phelan-McDermid syndrome. It is for children aged 3 to 12 who have a specific genetic change (SHANK3) and weigh at least 10 kg.
This study follows children who completed the RB001 gene therapy trial for Phelan-McDermid syndrome (caused by changes in the SHANK3 gene) to monitor their health and progress over a longer period of time.
This study tests whether a medication called NNZ-2591 can help children with Phelan-McDermid syndrome, a rare genetic condition that affects brain development and learning. Researchers want to see if this drug can improve how children with this condition develop and learn.
This study looks at families with Phelan-McDermid syndrome, a condition linked to changes in the SHANK3 gene. It aims to find other genes that might influence how the syndrome affects a person, by collecting genetic information from patients and their family members.
This early-phase study tests a gene therapy meant to treat people who have IGHMBP2 gene changes. It aims to improve the way the nervous system works and to see if the treatment is safe, especially in young children.
This trial tests a one-time gene therapy (BFB-101) for children with a rare genetic condition called SPG47, which causes problems with movement and development. The therapy aims to replace the faulty gene to help improve symptoms.
Hear when a new Phelan-McDermid Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.