Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Recruiting now · Phase 1
Conditions studied: RPS19 Deficient Diamond-Blackfan Anemia
In brief
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. The main questions it aims to answer are: * Is APR-2020 safe and well tolerated? * Does APR-2020 modify or correct an underlying genetic condition which causes DBA? * Does APR-2020 reduce or eliminate the need for blood transfusions and/or restore certain blood counts affected by DBA? Participants will: * Take the drug one time as an infusion. * Undergo two rounds of a cellular harvest procedure in which their own cells will be used in the manufacturing of their own participant-specific product. * Initially return to the clinic for two years of follow up at increasingly sparse intervals.
Key facts
- Study ID
- NCT07476183
- Run by
- Apriligen, Inc.
- People needed
- 4
- Starts
- 2026-04-16
- Expected to finish
- 2028-09-01
- Last updated by the study team
- 2026-07-21
Who can join
Age: 2 and older, up to 25. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Stanford University - Stanford Children's Health — Stanford, California, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
Full record on ClinicalTrials.gov
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