22 completed, 6 terminated, 5 recruiting, 2 active not recruiting. 38 Phase 2, 17 Phase 1, 2 Phase 3.
Follow Fanconi Anemia: new trials by email Literature Review of Fanconi Anemia trials Research Report on Fanconi Anemia
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 |
| 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| A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia FP-045 - Fanconi Anemia | Phase 1 / Phase 2 | Withdrawn | Foresee Pharmaceuticals Co., Ltd.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 | |
| Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant - Fanconi Anemia | Phase 1 / Phase 2 | Recruiting | Porteus, Matthew, MD | 18 | 2021-12-07 | 2026-01-27 |
| Eltrombopag for Treatment of Fanconi Anemia Eltrombopag - Fanconi Anemia | Phase 2 | Completed | Julian Sevilla | 8 | 2020-12-02 | 2023-09-21 |
| 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 |
| Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant Abatacept - Graft Versus Host Disease | Phase 2 | Active not recruiting | Emory University | 30 | 2019-08-22 | 2026-02-25 |
| 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 |
| Eltrombopag for Fanconi Anemia Eltrombopag - Fanconi Anemia | Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 25 | 2018-11-02 | 2026-09-16 |
| A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia Busulfan, Fludarabine, Cyclophosphamide - Fanconi Anemia, Myelodysplastic Syndrome (MDS) | Phase 2 | Terminated | Memorial Sloan Kettering Cancer Center | 3 | 2018-05-15 | 2022-04-12 |
| Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia Quercetin (dietary supplement) - Fanconi Anemia, Squamous Cell Carcinoma | Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 48 | 2018-05-08 | 2026-04-20 |
| Pilot Study of Metformin for Patients With Fanconi Anemia metformin HCl - Fanconi Anemia | Phase 2 | Completed | Boston Children's Hospital | 15 | 2018-03-29 | 2022-11-29 |
| Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor G-CSF, Plerixafor - Fanconi Anemia | Phase 1 / Phase 2 | Completed | Assistance Publique - Hôpitaux de Paris | 4 | 2017-02-10 | 2025-09-11 |
| Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study Rimiducid, rivogenlecleucel - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Terminated | Bellicum Pharmaceuticals | 187 | 2017-01-01 | 2023-09-26 |
| A Study of LY2606368 (Prexasertib) in Patients With Solid Tumors With Replicative Stress or Homologous Repair Deficiency LY2606368 - Advanced Cancers | Phase 2 | Completed | Dana-Farber Cancer Institute | 27 | 2016-10-12 | 2022-10-21 |
| A Phase 1/2 Trial of SRA737 in Subjects With Advanced Cancer SRA737 - Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL) | Phase 1 / Phase 2 | Completed | Sierra Oncology LLC - a GSK company | 107 | 2016-07-01 | 2023-06-18 |
| A Phase 1/2 Trial SRA737 in Combination With Gemcitabine and Cisplatin or Gemcitabine Alone in Advanced Cancer Subjects SRA737, gemcitabine, cisplatin, SRA737, gemcitabine - Advanced Solid Tumors | Phase 1 / Phase 2 | Completed | Sierra Oncology LLC - a GSK company | 153 | 2016-07-01 | 2023-06-22 |
| Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs), Genetically Engineered Hematopoietic Stem/Progenitor Cells, Laboratory Biomarker Analysis - Fanconi Anemia | Phase 1 / Phase 2 | Completed | Hospital Infantil Universitario Niño Jesús, Madrid, Spain | 9 | 2016-01-07 | 2024-03-21 |
| Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant BPX-501 T cells, Rimiducid - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Terminated | Bellicum Pharmaceuticals | 187 | 2014-04-01 | 2023-09-29 |
| HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy Busulfan, Cyclophosphamide, Fludarabine - Fanconi Anemia, Severe Marrow Failure | Phase 2 | Recruiting | Children's Hospital Medical Center, Cincinnati | 70 | 2014-04-01 | 2025-11-12 |
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Anemia, Aplastic (8) · Myelodysplastic Syndromes (8) · Leukemia (6) · Leukemia, Myeloid, Acute (5) · Anemia, Diamond-Blackfan (4) · Bone Marrow Failure Disorders (4) · Dyskeratosis Congenita (4) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (4) · Anemia, Sickle Cell (3) · Cytopenia (3) · Hematologic Diseases (3) · Leukemia, Myeloid, Chronic-Phase (3) · Lymphoma, Non-Hodgkin (3) · Primary Immunodeficiency Diseases (3)
Bone Marrow Diseases (38) · Hemic and Lymphatic Diseases (38) · Metabolic Diseases (38) · Nutritional and Metabolic Diseases (38) · Anemia, Hypoplastic, Congenital (37) · Congenital Bone Marrow Failure Syndromes (37) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (37) · DNA Repair-Deficiency Disorders (37) · Genetic Diseases, Inborn (37) · Anemia (36) · Hematologic Diseases (35) · Bone Marrow Failure Disorders (34) · Anemia, Aplastic (30) · Neoplasms by Histologic Type (12) · Neoplasms (11)
Cyclophosphamide (15) · fludarabine (11) · fludarabine phosphate (11) · Antilymphocyte Serum (9) · Busulfan (8) · Filgrastim (8) · Granulocyte Colony-Stimulating Factor (7) · Methylprednisolone (6) · Mycophenolic Acid (5) · Whole-Body Irradiation (5) · Alemtuzumab (4) · Cyclosporine (4) · Radiotherapy (4) · plerixafor (4) · Melphalan (3)
Masonic Cancer Center, University of Minnesota (5) · Children's Hospital Medical Center, Cincinnati (3) · National Heart, Lung, and Blood Institute (NHLBI) (3) · Bellicum Pharmaceuticals (2) · Boston Children's Hospital (2) · Sierra Oncology LLC - a GSK company (2) · Agnieszka Czechowicz (1) · Assistance Publique - Hôpitaux de Paris (1) · Baylor College of Medicine (1) · Columbia University (1) · Dana-Farber Cancer Institute (1) · Emory University (1) · Foresee Pharmaceuticals Co., Ltd. (1) · Fred Hutchinson Cancer Center (1) · Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau (1)
United States (26) · Spain (5) · United Kingdom (3) · Italy (2) · Brazil (1) · France (1) · Germany (1)
Margaret MacMillan, MD (2) · Akiko Shimamura, MD, PhD (1) · Andre Larochelle, M.D. (1) · Colin A Sieff, MB.BCh (1) · David A Margolis, MD (1) · David A. Williams (1) · Farid Boulad, MD (1) · Geoffrey Shapiro, MD, PhD (1) · Hans-Peter Kiem (1) · Heather J Symons, MD, MHS (1) · James Garvin, MD, PhD (1) · Joanne Kurtzberg (1) · John E. Wagner, Jr. (1)
PMID 25824692, PMID 18723429, PMID 31501599, PMID 33159275, PMID 34485595, PMID 34665631, PMID 34751952, PMID 35500223, PMID 36378548, PMID 37120671, PMID 39642902, PMID 40665878, PMID 40696207, PMID 40859225
2014 (1), P30CA015704 (1), R01HL081499 (1)