Clinical trials
Metachromatic Leukodystrophy clinical trials
Below are recruiting metachromatic leukodystrophy clinical trials, each written for real people, not researchers. We’re tracking 7 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT04925349Recruiting
Study blood immune patterns in several rare brain diseases
This study looks at immune cells in blood to better understand rare brain conditions caused by genetic changes. It may help researchers understand why symptoms happen and how the immune system is involved.
Le Kremlin-BicêtreAges 1.3 years–60 years - 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 - 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 - NCT03725670Recruiting
Gene therapy injection for metachromatic leukodystrophy (MLD)
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.
Shenzhen, GuangdongAges 1 month–50 years - NCT07046338Recruiting
Gene therapy for MLD using the patient's own stem cells
This trial tests a new gene therapy that uses a patient's own blood stem cells to treat MLD (metachromatic leukodystrophy), a rare genetic disorder that damages the nervous system. The goal is to see if this treatment can stop or slow the disease.
Shenzhen, GuangdongAges 1 month–50 years - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT05368038Enrolling by invitation
Newborn screening program for babies up to 4 weeks old
This trial tests a flexible newborn screening process that looks for certain conditions early in life. It may help by catching problems sooner, when treatment can be started earlier.
Brooklyn, New YorkAges Up to 4 weeks
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Common questions
- Are there clinical trials for metachromatic leukodystrophy?
- Yes. Clin2 currently lists 7 recruiting metachromatic leukodystrophy 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 metachromatic leukodystrophy 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 metachromatic leukodystrophy 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.