Scientists unravel genetic secrets of bone marrow failure
NCT ID NCT07102849
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study aims to understand the genetic and molecular changes in people with bone marrow failure disorders. Researchers will analyze blood and bone marrow samples from up to 1,400 participants to track how these changes evolve over time. The goal is to better predict disease outcomes and identify new targets for future treatments.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this research could reveal why some patients develop more serious blood cancers, pointing toward better monitoring or prevention strategies.
- What could go wrong
- This is an observational study, not a treatment trial. It may not directly lead to new therapies, and findings may not apply to all patients.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 1,400 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Sep 2025
- Expected to finish
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Jul 2030
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
This study will utilize biospecimens and data with identifiers from up to 1,400 research participants with BMFS, aged 2 years and older, alongside age matched healthy volunteers aged 8 years and older.
- Ages
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2 to 120 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* This study will utilize biospecimens and data with identifiers from up to 1,400 research participants with BMFS, aged 2 years and older, alongside age matched healthy volunteers aged 8 years and older.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
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