3 recruiting. 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Recruiting 2 |
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data Inherited Bone Marrow Failure Syndrome, Fanconi Anemia | Recruiting | Istituto Giannina Gaslini | 250 | 2025-05-29 | 2026-10-16 | |
| Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures Alemtuzumab, Fludarabine, Melphalan - Primary Immune Deficiency Disorder, Immune Deficiency Disease | Phase 2 | Recruiting | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 27 | 2020-02-12 | 2025-11-28 |
| Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU) - Fanconi Anemia, Severe Aplastic Anemia | Phase 2 | Recruiting | Masonic Cancer Center, University of Minnesota | 48 | 2018-11-13 | 2026-01-30 |
Dyskeratosis Congenita (3) · Fanconi Anemia (3) · Bone Marrow Failure Disorders (2) · Anemia, Aplastic (1) · Congenital Bone Marrow Failure Syndromes (1) · Congenital amegakaryocytic thrombocytopenia (1) · Hemoglobinuria, Paroxysmal (1) · Hoyeraal Hreidarsson syndrome (1) · Immune Deficiency Disease (1) · Myelodysplastic Syndromes (1) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (1) · Primary Immunodeficiency Diseases (1)
Anemia (3) · Anemia, Hypoplastic, Congenital (3) · Bone Marrow Diseases (3) · Congenital Abnormalities (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · DNA Repair-Deficiency Disorders (3) · Genetic Diseases, Inborn (3) · Genetic Diseases, X-Linked (3) · Hematologic Diseases (3) · Hemic and Lymphatic Diseases (3) · Metabolic Diseases (3) · Nutritional and Metabolic Diseases (3) · Skin Abnormalities (3) · Skin Diseases (3) · Skin Diseases, Genetic (3)
Alemtuzumab (2) · Cyclophosphamide (2) · Melphalan (2) · Whole-Body Irradiation (2) · fludarabine (2) · Busulfan (1) · Granulocyte Colony-Stimulating Factor (1) · Methylprednisolone (1) · Mycophenolic Acid (1) · Rituximab (1) · Tacrolimus (1)
Istituto Giannina Gaslini (1) · Masonic Cancer Center, University of Minnesota (1) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (1)
United States (2) · Italy (1)
Heather J Symons, MD, MHS (1) · Margaret MacMillan, MD, Msc, FRCPC (1)