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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)

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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: Not yet recruiting
Phase of Trial: Phase I

Latest Information Update: 25 Mar 2026

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At a glance

  • Drugs APR 2020 (Primary)
  • Indications Diamond-Blackfan syndrome
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors Apriligen

Most Recent Events

  • 25 Mar 2026 New trial record

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