Trial Profile

A Phase 1, Open-label, Single-arm Study of APR 2020 in Transfusion Dependent, Steroid Resistant Pediatric and Adolescent Subjects With RPS19 Deficient Diamond Blackfan Anemia by Transplantation of Autologous CD34+ Stem Cells Transduced With CLIN LV EFS coRPS19 PRE* (APR-2020)

Status: Recruiting

Phase of Trial: Phase I

Latest Information Update: 17 May 2026

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At a glance

20 Apr 2026 Planned primary completion date changed from 1 Mar 2027 to 1 Apr 2027.
20 Apr 2026 Status changed from not yet recruiting to recruiting.
25 Mar 2026 New trial record

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