Gene therapy trial for children with ML4
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy given by a one-time injection into the spinal fluid for children with Mucolipidosis type IV. It aims to see if the therapy is safe and can help slow or stop the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age between 1.5 and 8 years old
- Diagnosed with Mucolipidosis type IV (ML4) caused by a change in the MCOLN1 gene
- Able to safely get a lumbar puncture (spinal tap) and sedation
- Up to date on childhood vaccines
- Not needing a breathing machine or extra oxygen for more than 12 hours a day
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 early-stage trial tests VGN-R09b, a gene therapy, given to children with AADC deficiency who are not doing well on standard medicines. It aims to improve symptoms and quality of life by addressing the underlying cause of AADC deficiency.
This trial tests a direct gene-therapy injection into the body to treat MLD, a genetic brain condition. It may help replace a faulty gene so the disease process slows or changes.
This trial tests a gene therapy for people with a specific mutation (S295L) in the SLC6A1 gene, which causes a neurodevelopmental disorder. The therapy aims to correct the genetic issue, and participants will be monitored closely for safety and effectiveness.
This study tests a new gene therapy called JWK008 for adults with MPS type I. The goal is to see if it is safe and can help improve symptoms. You may be able to join if you are 18 or older and have not had serious side effects from enzyme replacement therapy.
This early-stage trial tests JR-446, a new treatment for MPS IIIB, a rare genetic disorder that affects the brain and body. The treatment is delivered directly into the fluid around the spinal cord and aims to slow or improve symptoms in young children.
This trial studies a one-time gene therapy meant to help children with SPG50 disease caused by changes in the AP4M1 gene. Researchers will give the treatment and closely monitor safety and early signs of benefit.
Hear when a new Mucolipidosis Type IV trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.