4 recruiting, 1 active not recruiting, 1 terminated. 2 Phase 1, 1 Not applicable, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Recruiting 1, Terminated 1 |
| Not applicable | 1 | Recruiting 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow Inherited Bone Marrow Failure Syndrome: new trials by email Literature Review of Inherited Bone Marrow Failure Syndrome trials Research Report on Inherited Bone Marrow Failure Syndrome
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 | |
| Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency TruSight Oncology, imatinib - Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies | Phase 1 | Recruiting | National Cancer Institute (NCI) | 71 | 2023-12-19 | 2026-10-30 |
| A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies Decitabine, Filgrastim - Acute Myeloid Leukemia, Myelodysplastic Syndromes | Phase 2 | Active not recruiting | Franziska Wachter | 29 | 2023-04-19 | 2026-08-20 |
| Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia Fanconi Anemia, Inherited Bone Marrow Failure Syndrome | Recruiting | National Cancer Institute (NCI) | 200 | 2023-03-23 | 2026-11-06 | |
| Pre-myeloid Cancer and Bone Marrow Failure Clinic Study Biospecimen Collection, Bone Marrow Biopsy, Punch Biopsy - Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome | Not applicable | Recruiting | Mayo Clinic | 2000 | 2017-01-16 | 2026-02-23 |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases BPX-501 and Rimiducid - Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis | Phase 1 | Terminated | Bellicum Pharmaceuticals | 1 | 2015-02-01 | 2024-03-25 |
Congenital Bone Marrow Failure Syndromes (6) · Fanconi Anemia (3) · Dyskeratosis Congenita (2) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Bone Marrow Failure Disorders (1) · Congenital amegakaryocytic thrombocytopenia (1) · Cytopenia (1) · Granulomatous Disease, Chronic (1) · Hematologic Neoplasms (1) · Hemoglobinopathies (1) · Hemoglobinuria, Paroxysmal (1) · Leukemia, Myeloid, Acute (1) · Lymphohistiocytosis, Hemophagocytic (1) · Metabolic Diseases (1)
Bone Marrow Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Hematologic Diseases (6) · Hemic and Lymphatic Diseases (6) · Infant, Newborn, Diseases (6) · Bone Marrow Failure Disorders (5) · Genetic Diseases, Inborn (4) · Anemia (3) · Anemia, Aplastic (3) · Anemia, Hypoplastic, Congenital (3) · DNA Repair-Deficiency Disorders (3) · Nutritional and Metabolic Diseases (3) · Anemia, Hemolytic (2) · Congenital Abnormalities (2) · Genetic Diseases, X-Linked (2)
Biopsy (1) · Decitabine (1) · Filgrastim (1) · Genetic Counseling (1) · Granulocyte Colony-Stimulating Factor (1) · Imatinib Mesylate (1) · Specimen Handling (1)
National Cancer Institute (NCI) (2) · Bellicum Pharmaceuticals (1) · Franziska Wachter (1) · Istituto Giannina Gaslini (1) · Mayo Clinic (1)
United States (5) · Italy (1)
Franziska Wachter, MD (1) · Lea C Cunningham, M.D. (1) · Lisa J McReynolds, M.D. (1) · Mrinal S. Patnaik, MBBS (1)