Study of a new drug for Pelizaeus Merzbacher disease in boys
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial is testing an experimental drug (ION356) given by a lumbar puncture to see if it is safe and how it works in boys with Pelizaeus Merzbacher disease (PMD). The drug is designed to target the genetic cause of PMD.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of PMD caused by a duplication of the PLP1 gene.
- You must be a boy between 2 and 17 years old.
- You must be able to have MRIs and lumbar punctures (spinal taps).
- You cannot have had gene therapy, cell transplantation, or experimental brain surgery before.
- You must not have had other investigational drugs or oligonucleotide treatments recently (within 1–12 months depending on type).
- You must be free of other medical conditions that could interfere with the study.
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 study follows children with Pelizaeus-Merzbacher disease (PMD) who have a specific genetic change (PLP1 duplication). It aims to learn which body measurements (“biomarkers”) and symptoms change over time, to better understand disease progression.
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 is building a large, detailed group of people—some with Parkinson’s and some at higher risk—to understand early changes over time. It uses brain imaging (DaTscan SPECT), sometimes spinal fluid tests (lumbar puncture), and genetic information to help researchers better measure disease and develop future treatments.
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.
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 study tests a gene therapy called IT MELPIDA for children with SPG50, a genetic condition that affects movement and development. The treatment is given through a lumbar puncture (spinal tap) and aims to slow or stop the loss of motor skills.
Hear when a new Pelizaeus-Merzbacher Disease trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.