Clinical trials
Pelizaeus-Merzbacher Disease clinical trials
Below are recruiting pelizaeus-merzbacher disease clinical trials, each written for real people, not researchers. We’re tracking 5 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT03047369Recruiting
Myelin brain disorder research biobank for samples and data
This study collects brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
Los Angeles, CaliforniaAges Any age - NCT03333200Recruiting
Study of genetic brain diseases over time
This trial follows people with genetic neurodegenerative (brain-wasting) disorders over time to better understand how the condition changes. The goal is to learn patterns that could help future treatments or care plans.
Pittsburgh, PennsylvaniaAges Any age - NCT05659901Recruiting
Study tracks symptoms and test results in PLP1-confirmed PMD
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.
Atlanta, GeorgiaAges 6 months–17 years - NCT06150716RecruitingPhase 1
Study of a new drug for Pelizaeus Merzbacher disease in boys
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.
Atlanta, GeorgiaAges 2–17 - NCT02254863RecruitingPhase 1
Stem cell transplant into the spinal fluid for rare brain diseases
This Phase 1 trial tests a transplant using special donated stem-cell–like cells placed into the spinal fluid to treat certain inherited brain diseases. It is mainly designed to check safety, and it may offer benefit by targeting brain and nerve damage.
Durham, North CarolinaAges 1 week–22 years
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Pelizaeus-Merzbacher Disease trials by city
Studies with a site in or near these metro areas.
Pelizaeus-Merzbacher Disease trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for pelizaeus-merzbacher disease?
- Yes. Clin2 currently lists 5 recruiting pelizaeus-merzbacher disease studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a pelizaeus-merzbacher disease trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a pelizaeus-merzbacher disease trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.