75 completed, 20 recruiting, 20 terminated, 6 active not recruiting. 66 Phase 2, 42 Phase 1, 19 Not applicable, 7 Phase 3, 1 Early Phase 1.
Follow Anemia, Hypoplastic, Congenital: new trials by email Literature Review of Anemia, Hypoplastic, Congenital trials Research Report on Anemia, Hypoplastic, Congenital
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 | 2026-11-09 | 2026-11-04 | |
| Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi Anemia Metformin, Naproxen Sodium - Fanconi Anemia, Head and Neck Cancer | Phase 1 | Recruiting | Rockefeller University | 80 | 2026-10-14 | 2026-10-28 |
| 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 | 2026-11-03 |
| Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS) Mobilization Regimen - Diamond Blackfan Anemia | Recruiting | Northwell Health | 10 | 2026-05-11 | 2026-11-03 | |
| 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 |
| 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Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients Nitisinone - Hereditary Tyrosinemia, Type I | Withdrawn | Swedish Orphan Biovitrum | 2025-09-01 | 2025-12-23 | ||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 |
| 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 |
| 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 | |
| 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 | |
| Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia Specimen Donation - Fanconi Anemia | Terminated | Sanguine Biosciences | 4 | 2023-05-24 | 2024-04-18 | |
| 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 | 2026-10-01 | |
| Microcephaly, Fanconi Anemia and Praxial Disorders MRI of the hand and forearm, - Fanconi Anemia | Not applicable | Withdrawn | Assistance Publique - Hôpitaux de Paris | 2023-01-30 | 2023-02-03 | |
| Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia Time-Restricted Feeding - Fanconi Anemia | Not applicable | Completed | Children's Hospital Medical Center, Cincinnati | 3 | 2022-11-11 | 2024-07-24 |
| Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia Metformin - Fanconi Anemia | Phase 3 | Unknown | Ain Shams University | 30 | 2022-10-05 | 2024-07-25 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 |
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Fanconi Anemia (95) · Anemia, Diamond-Blackfan (56) · Anemia, Sickle Cell (32) · Anemia, Aplastic (31) · Thalassemia (25) · Myelodysplastic Syndromes (21) · Bone Marrow Failure Disorders (17) · Shwachman-Diamond Syndrome (16) · Dyskeratosis Congenita (15) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (15) · Leukemia, Myeloid, Acute (13) · Hemoglobinopathies (12) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (12) · Leukemia (10) · Lymphoma, Non-Hodgkin (10)
Bone Marrow Diseases (137) · Hemic and Lymphatic Diseases (136) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (135) · Genetic Diseases, Inborn (131) · Anemia (129) · Congenital Bone Marrow Failure Syndromes (129) · Hematologic Diseases (128) · Bone Marrow Failure Disorders (120) · Nutritional and Metabolic Diseases (114) · Metabolic Diseases (111) · Anemia, Aplastic (106) · DNA Repair-Deficiency Disorders (98) · Red-Cell Aplasia, Pure (48) · Anemia, Hemolytic (42)
Cyclophosphamide (26) · fludarabine phosphate (24) · fludarabine (20) · Antilymphocyte Serum (16) · Busulfan (15) · Whole-Body Irradiation (12) · Alemtuzumab (11) · Filgrastim (11) · Granulocyte Colony-Stimulating Factor (10) · Melphalan (10) · Methylprednisolone (10) · Cyclosporine (9) · Mycophenolic Acid (9) · Peripheral Blood Stem Cell Transplantation (9) · Bone Marrow Transplantation (6)
Masonic Cancer Center, University of Minnesota (10) · National Heart, Lung, and Blood Institute (NHLBI) (9) · Children's Hospital Medical Center, Cincinnati (7) · Fred Hutchinson Cancer Center (6) · University of California, San Francisco (6) · Novartis Pharmaceuticals (5) · Assistance Publique - Hôpitaux de Paris (4) · National Cancer Institute (NCI) (4) · Roswell Park Cancer Institute (4) · Bellicum Pharmaceuticals (3) · Boston Children's Hospital (3) · Emory University (3) · Fairview University Medical Center (3) · Memorial Sloan Kettering Cancer Center (3) · Northwell Health (3)
United States (105) · Spain (11) · United Kingdom (9) · Italy (8) · Germany (6) · China (5) · France (5) · Belgium (4) · Egypt (3) · Greece (3) · Netherlands (3) · Taiwan (3) · Thailand (3) · Turkey (Türkiye) (3) · Australia (2)
Adrianna Vlachos, MD (3) · Farid Boulad, MD (3) · Hans-Peter Kiem (3) · Margaret MacMillan, MD (3) · Paul Szabolcs, MD (3) · Daniel J. Weisdorf (2) · Diane George, MD (2) · Elliott Vichinsky, MD (2) · George Chen (2) · Grover C. Bagby, MD (2) · Janel Long-Boyle, PharmD, PhD (2) · Janel R Long-Boyle, PharmD, PhD (2) · John E. Wagner, Jr. (2)
PMID 25824692, PMID 39642902, PMID 18723429, PMID 19951979, PMID 19996412, 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 31151984, PMID 31501599, PMID 31836590, PMID 32015000, PMID 32634238, PMID 32750127, PMID 32790018, PMID 33025707, PMID 33159275, PMID 33810313, PMID 34162668, PMID 34485595, PMID 34665631, PMID 34751952, PMID 34852175, PMID 35322185, PMID 35360980, PMID 35500223, PMID 35726608, PMID 36378548, PMID 37120671, PMID 37142543, PMID 37354000, PMID 39279436, PMID 39562502, PMID 39820512, PMID 40151166
P30CA015704 (3), R01HL081499 (3), 2004 (2), R01DK057778 (2), 2014 (1), P01HL048546 (1), P30CA016672 (1), P30CA069533 (1), U10HL069294 (1)