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Clinical trial

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)

NCT07476183 is registered in ClinicalTrials.gov with Apriligen, Inc. as lead sponsor.

NCT NCT07476183
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Registry record

As filed in ClinicalTrials.gov.

Title
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)
Lead sponsor
Apriligen, Inc.
Phase
Phase 1
Overall status
Recruiting
Start date
April 16, 2026
Completion date
September 30, 2028
Enrollment
4 (estimated)
Sites
2

Conditions

As the registry lists them; a condition with its own page is linked.

rps19 deficient diamond-blackfan anemia