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Gene therapy may free kids with rare anemia from lifelong blood transfusions

NCT ID NCT07476183

Recruiting now Disease control Sponsor: Apriligen, Inc. Source: ClinicalTrials.gov ↗

First seen Mar 26, 2026 · Last updated Apr 30, 2026 · Updated 5 times

Summary

This early-stage trial tests a gene therapy called APR-2020 in children and teens with a specific type of Diamond-Blackfan anemia (DBA). The treatment uses the patient's own blood stem cells, modified to correct the genetic defect, and given as a one-time infusion. The goal is to see if it is safe and can reduce or eliminate the need for regular blood transfusions. Only 4 participants aged 2 to 18 are being enrolled.

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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: •••••@•••••

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02215, United States

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

  • Stanford University - Stanford Children's Health

    NOT_YET_RECRUITING

    Stanford, California, 94304, United States

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

Conditions

Explore the condition pages connected to this study.

RPS19 DEFICIENT DIAMOND-BLACKFAN ANEMIA