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

U.S., March 18 -- ClinicalTrials.gov registry received information related to the study (NCT07476183) titled 'Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia' on March 12.

Brief 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: ...