Condition
Inborn Errors of Metabolism clinical trials and research
As of November 12, 2026, Find a Trial lists 5 Inborn Errors of Metabolism clinical trials that are recruiting, at sites in 3 countries (most in United States (3), Argentina (1), Belgium (1)). By phase: Early Phase 1 (1), Phase 1/2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 5Clinical trials
- 3Countries
Newest Inborn Errors of Metabolism clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Using CD45RA Depleted Stem Cells to Prevent Infections After Transplant | Phase 1/2 | Philadelphia, Pennsylvania | Recruiting | Feb 21, 2025 |
| Registry for Rare Diseases | — | Buenos Aires, Buenos Aires | Recruiting | Aug 27, 2024 |
| Screening for rare diseases using blood and urine samples | — | Brussels and 3 other locations | Recruiting | Apr 11, 2024 |
| Access to stem cell transplant for patients without matched donors | Early Phase 1 | Philadelphia, Pennsylvania | Recruiting | Feb 5, 2015 |
| Observational study of Methylmalonic Acidemia | — | Washington D.C., District of Columbia and 2 other locations | Recruiting | Feb 19, 2004 |
All recruiting trials (5)
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Using CD45RA Depleted Stem Cells to Prevent Infections After Transplant
This study is testing whether using special stem cells can help patients with high-risk leukemia recover their immune system faster and prevent infections after a stem cell transplant.
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Registry for Rare Diseases
This study is creating a detailed registry to collect information on rare diseases like amyloidosis and sarcoidosis to help improve how they are diagnosed and treated.
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Screening for rare diseases using blood and urine samples
This study is trying to collect blood and urine samples from people with suspected rare diseases and healthy individuals to see if they can find specific markers that help diagnose conditions like metabolic disorders and autism.
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Access to stem cell transplant for patients without matched donors
This study is testing a new way to help patients without a matched donor get a stem cell transplant by using a special device to reduce complications and improve their chances of recovery.
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Observational study of Methylmalonic Acidemia
This study looks at how often complications like metabolic stroke and developmental delays happen in people of all ages with Methylmalonic Acidemia to help improve future treatments.
Related topics
Quick answers
How many Inborn Errors of Metabolism 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 3 countries. The most sites are in United States (3 trials), Argentina (1 trial), Belgium (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Early Phase 1: 1, Phase 1/2: 1. 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.