Condition
Osteopetrosis clinical trials and research
As of November 12, 2026, Find a Trial lists 2 Osteopetrosis clinical trials that are recruiting, at sites in 2 countries (most in Italy (1), United States (1)). By phase: Phase 1/2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 2Clinical trials
- 2Countries
All recruiting trials (2)
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Gene-modified stem cell therapy for children with autosomal recessive osteopetrosis
This study tests a gene-modified stem cell therapy (FT024) in eight young children with TCIRG1-related autosomal recessive osteopetrosis to see if it is safe and feasible and to observe potential benefits.
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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.
Related topics
Quick answers
How many Osteopetrosis clinical trials are recruiting right now?
2, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 2 countries. The most sites are in Italy (1 trial), United States (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
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.