Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Recruiting · NCT07476183 · Registry updated 2026-07-21Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, pure red-cell aplasia | Recruiting | Phase 1 | 2026-07-21 | NCT07476183 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Apriligen, Inc. | 1 |
2 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Boston Children's Hospital | Boston | United States | 20 |
| Stanford University - Stanford Children's Health | Stanford | United States | 1 |