Condition: RPS19 Deficient Diamond-Blackfan Anemia · Sponsor: Apriligen, Inc.
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.
This description comes directly from the study's public registry record.
Nova Silver · 617-306-3020 · nsilver@apriligen.com
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| Stanford University - Stanford Children's Health | Stanford, California, United States | Recruiting |
| Boston Children's Hospital | Boston, Massachusetts, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07476183