Clinical Trial

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

Recruiting Phase 1

Summary

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.

Protocol Amendment History 3 changes

critical Primary endpoint(s) modified 2026-07-22

critical Recruitment opened 2026-04-21

notable Primary completion pushed: 2027-03 -> 2027-04 2026-04-21

Trial Details

NCT Number	NCT07476183
Lead Sponsor	Apriligen, Inc.
Conditions	RPS19 Deficient Diamond-Blackfan Anemia
Enrollment	4 participants
Start Date	2026-04-16
Primary Completion	2027-04 (estimated)
Study Completion	2028-09 (estimated)
Updated on ClinicalTrials.gov	2026-07-21