Condition
Pelizaeus-Merzbacher Disease clinical trials and research
As of November 12, 2026, Find a Trial lists 5 Pelizaeus-Merzbacher Disease clinical trials that are recruiting, at sites in 8 countries (most in United States (5), France (2), Germany (2)). By phase: Phase 1 (2). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 5Clinical trials
- 8Countries
Newest Pelizaeus-Merzbacher Disease clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Evaluating a new treatment for Pelizaeus-Merzbacher Disease in children | Phase 1 | Atlanta, Georgia and 6 other locations | Recruiting | Nov 29, 2023 |
| Characterizing biomarkers and disease progression in Pelizaeus-Merzbacher Disease | — | Atlanta, Georgia and 9 other locations | Recruiting | Dec 21, 2022 |
| Long-term observation of rare genetic brain disorders | — | Pittsburgh, Pennsylvania | Recruiting | Nov 6, 2017 |
| Collecting data and samples from patients with leukodystrophy | — | Los Angeles, California and 22 other locations | Recruiting | Feb 9, 2017 |
| Using umbilical cord blood cells to treat inherited metabolic diseases | Phase 1 | Durham, North Carolina | Recruiting | Oct 2, 2014 |
All recruiting trials (5)
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Evaluating a new treatment for Pelizaeus-Merzbacher Disease in children
This study is testing a new drug called ION356 to see if it is safe and tolerable for children with Pelizaeus-Merzbacher Disease who have a specific genetic change.
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Characterizing biomarkers and disease progression in Pelizaeus-Merzbacher Disease
This study is trying to see how certain markers in the body change over time in people with Pelizaeus-Merzbacher Disease to help develop better treatments in the future.
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Long-term observation of rare genetic brain disorders
This study is tracking how rare genetic brain disorders progress over time to better understand their effects on patients.
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Collecting data and samples from patients with leukodystrophy
This study is collecting health information and samples from people with leukodystrophy to help find new causes and better treatments for the condition.
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Using umbilical cord blood cells to treat inherited metabolic diseases
This study is testing if a new treatment using special cells from umbilical cord blood can help people with inherited metabolic diseases and early signs of nerve damage feel better when combined with standard cord blood transplants.
Related topics
Quick answers
How many Pelizaeus-Merzbacher Disease clinical trials are recruiting right now?
5, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 8 countries. The most sites are in United States (5 trials), France (2 trials), Germany (2 trials), Netherlands (2 trials), Israel (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 1: 2. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.