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20-Year study aims to unlock secrets of rare bone marrow diseases

NCT ID NCT05012111

First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 30 times

Summary

This study follows 1,000 people with bone marrow failure diseases for up to 20 years to learn how the diseases and treatments affect the body over time. Participants aged 2 and older, including family members, will have regular check-ups, blood tests, and other exams. The goal is to better understand long-term outcomes, not to test a new treatment.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.