80 completed, 30 recruiting, 22 terminated, 9 active not recruiting. 72 Phase 2, 46 Phase 1, 23 Not applicable, 9 Phase 3, 1 Early Phase 1.
Follow Congenital Bone Marrow Failure Syndromes: new trials by email Literature Review of Congenital Bone Marrow Failure Syndromes trials Research Report on Congenital Bone Marrow Failure Syndromes
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 |
|---|---|---|---|---|---|---|
| Prenatal Transplantation for Fetuses With Fanconi Anemia IUHSCT for FA-affected fetuses - Fanconi Anemia, Anemia, Hypoplastic, Congenital | Phase 1 / Phase 2 | Not yet recruiting | Agnieszka Czechowicz | 12 | 2028-01-01 | 2026-06-16 |
| Clinical Genetics Branch Eligibility Screening Survey Melanoma, Li-Fraumeni Syndrome | Not yet recruiting | National Cancer Institute (NCI) | 1000 | 2026-10-27 | 2026-10-22 | |
| MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients MRIs - Fanconi Anemia | Recruiting | University of Minnesota | 80 | 2026-06-23 | 2026-07-02 | |
| FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector Gene-modified autologous stem cells - Fanconi Anemia | Not applicable | Terminated | Shenzhen Geno-Immune Medical Institute | 10 | 2026-06-01 | 2026-08-24 |
| Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS) Mobilization Regimen - Diamond Blackfan Anemia | Recruiting | Northwell Health | 10 | 2026-06-01 | 2026-04-27 | |
| Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD) Quercetin - Dyskeratosis Congenita, Telomere Disease | Phase 1 | Recruiting | Children's Hospital Medical Center, Cincinnati | 12 | 2026-05-29 | 2026-09-10 |
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia APR-2020 - RPS19 Deficient Diamond-Blackfan Anemia | Phase 1 | Recruiting | Apriligen, Inc. | 4 | 2026-04-16 | 2026-07-21 |
| Nucleoside Therapy in Patients With Telomere Biology Disorders deoxycytidine, deoxythymidine - Telomere Biology Disorders, Dyskeratosis Congenita | Phase 1 | Recruiting | Suneet Agarwal | 36 | 2025-09-29 | 2026-08-04 |
| A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients Nitisinone - Hereditary Tyrosinemia, Type I | Withdrawn | Swedish Orphan Biovitrum | 2025-09-01 | 2025-12-23 | ||
| 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 | |
| Corrective Exercise and Rehabilitation in Fanconi Anemia: A Case Study Corrective exercise and rehabilitation - Fanconi Anemia | Not applicable | Completed | Pardis Specialized Wellness Institute | 1 | 2025-05-03 | 2025-08-12 |
| Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3 - Leukemia, High Risk Acute Lymphoblastic Leukemia | Phase 1 / Phase 2 | Recruiting | Children's Hospital of Philadelphia | 100 | 2025-03-21 | 2026-04-15 |
| Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia Fanconi Anemia | Active not recruiting | National Cancer Institute (NCI) | 20 | 2024-12-05 | 2026-10-02 | |
| Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC) Afatinib - Fanconi Anemia, Head and Neck Squamous Cell Carcinoma | Phase 1 / Phase 2 | Recruiting | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | 25 | 2024-11-08 | 2026-05-27 |
| A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections Mavorixafor, Placebo - Neutropenia | Phase 3 | Recruiting | X4 Pharmaceuticals | 176 | 2024-06-06 | 2026-08-06 |
| Prognostic Significance of Mutation Type and Chromosome Fragility in Fanconi Anemia Fanconi Anemia | Completed | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | 227 | 2024-05-16 | 2024-07-08 | |
| Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform Shwachman-Diamond Syndrome, SDS | Recruiting | Shwachman-Diamond Syndrome Alliance Inc | 8000 | 2024-02-07 | 2025-05-31 | |
| 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 | 2026-10-20 |
| Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia Bitopertin - Steroid-refractory Diamond-Blackfan Anemia (DBA) | Phase 1 / Phase 2 | Completed | National Heart, Lung, and Blood Institute (NHLBI) | 15 | 2023-07-25 | 2026-06-30 |
| A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia FP-045 - Fanconi Anemia | Phase 1 / Phase 2 | Withdrawn | Foresee Pharmaceuticals Co., Ltd. | 2023-06-30 | 2025-03-06 |
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Fanconi Anemia (94) · Anemia, Diamond-Blackfan (56) · Anemia, Aplastic (36) · Anemia, Sickle Cell (32) · Thalassemia (25) · Bone Marrow Failure Disorders (24) · Myelodysplastic Syndromes (23) · Dyskeratosis Congenita (22) · Shwachman-Diamond Syndrome (21) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (17) · Leukemia, Myeloid, Acute (14) · Hemoglobinopathies (13) · Leukemia (12) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (12)
Bone Marrow Diseases (157) · Hemic and Lymphatic Diseases (156) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (153) · Hematologic Diseases (147) · Genetic Diseases, Inborn (141) · Anemia (135) · Bone Marrow Failure Disorders (133) · Anemia, Hypoplastic, Congenital (132) · Nutritional and Metabolic Diseases (119) · Metabolic Diseases (115) · Anemia, Aplastic (105) · DNA Repair-Deficiency Disorders (97) · Red-Cell Aplasia, Pure (48) · Anemia, Hemolytic (43)
Cyclophosphamide (28) · fludarabine phosphate (27) · fludarabine (23) · Antilymphocyte Serum (18) · Busulfan (15) · Alemtuzumab (14) · Whole-Body Irradiation (14) · Filgrastim (12) · Granulocyte Colony-Stimulating Factor (11) · Methylprednisolone (11) · Cyclosporine (10) · Melphalan (10) · Mycophenolic Acid (10) · Peripheral Blood Stem Cell Transplantation (9) · Stem Cell Transplantation (8)
Masonic Cancer Center, University of Minnesota (12) · National Heart, Lung, and Blood Institute (NHLBI) (11) · Children's Hospital Medical Center, Cincinnati (8) · Fred Hutchinson Cancer Center (6) · National Cancer Institute (NCI) (6) · University of California, San Francisco (6) · Boston Children's Hospital (5) · Novartis Pharmaceuticals (5) · Assistance Publique - Hôpitaux de Paris (4) · Roswell Park Cancer Institute (4) · Bellicum Pharmaceuticals (3) · Emory University (3) · Fairview University Medical Center (3) · Memorial Sloan Kettering Cancer Center (3) · Northwell Health (3)
United States (121) · Spain (12) · United Kingdom (10) · Italy (9) · Germany (7) · Australia (6) · France (6) · Belgium (5) · China (5) · Egypt (4) · Greece (4) · Thailand (4) · Turkey (Türkiye) (4) · Canada (3) · Netherlands (3)
Adrianna Vlachos, MD (3) · Farid Boulad, MD (3) · Hans-Peter Kiem (3) · Margaret MacMillan, MD (3) · Parinda Mehta, MD (3) · Paul Szabolcs, MD (3) · Sharon A Savage, M.D. (3) · Daniel J. Weisdorf (2) · David J Young, M.D. (2) · Diane George, MD (2) · Elliott Vichinsky, MD (2) · George Chen (2) · Grover C. Bagby, MD (2)
PMID 25824692, PMID 39642902, PMID 18723429, PMID 19951979, PMID 19996412, PMID 20383216, PMID 20566896, PMID 21071497, PMID 23585526, PMID 24296492, PMID 24370862, PMID 24385534, PMID 24786393, PMID 25304614, PMID 25345697, PMID 25405500, PMID 25425683, PMID 25459642, PMID 25691036, PMID 25963299, PMID 26095669, PMID 27537786, PMID 28280134, PMID 28684371, PMID 30047424, PMID 31151984, PMID 31501599, PMID 31836590, PMID 32015000, PMID 32634238, PMID 32750127, PMID 32790018, PMID 33025707, PMID 33159275, PMID 33810313, PMID 34086408, PMID 34162668, PMID 34485595, PMID 34665631, PMID 34751952, PMID 34852175, PMID 34857525, PMID 35322185, PMID 35360980, PMID 35500223, PMID 35726608, PMID 36378548, PMID 37120671, PMID 37142543, PMID 37354000
P30CA015704 (3), R01HL081499 (3), 2004 (2), R01DK057778 (2), 2014 (1), 2023 (1), P01HL048546 (1), P30CA016672 (1), P30CA069533 (1), U10HL069294 (1)