| Not Yet Recruiting | Prenatal Transplantation for Fetuses With Fanconi Anemia NCT07408583 | Agnieszka Czechowicz | Phase 1 / Phase 2 |
| Recruiting | Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia NCT06744283 | National Cancer Institute (NCI) | — |
| Not Yet Recruiting | Clinical Genetics Branch Eligibility Screening Survey NCT07005297 | National Cancer Institute (NCI) | — |
| Completed | Corrective Exercise and Rehabilitation in Fanconi Anemia: A Case Study NCT06958380 | Pardis Specialized Wellness Institute | N/A |
| Recruiting | Afatinib in Patients with Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC) NCT06648096 | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | Phase 1 / Phase 2 |
| Completed | Prognostic Significance of Mutation Type and Chromosome Fragility in Fanconi Anemia NCT06490510 | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | — |
| Withdrawn | A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia NCT04522375 | Foresee Pharmaceuticals Co., Ltd. | Phase 1 / Phase 2 |
| Terminated | Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia NCT05910853 | Sanguine Biosciences | — |
| Recruiting | Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia NCT05687149 | National Cancer Institute (NCI) | — |
| Withdrawn | Microcephaly, Fanconi Anemia and Praxial Disorders NCT04656171 | Assistance Publique - Hôpitaux de Paris | N/A |
| Completed | Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia NCT05598515 | Children's Hospital Medical Center, Cincinnati | N/A |
| Unknown | Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia NCT06519786 | Ain Shams University | Phase 3 |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Recruiting | Role of Acetaldehyde in the Development of Oral Cancer NCT05973656 | Masonic Cancer Center, University of Minnesota | N/A |
| Enrolling By Invitation | Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 NCT07527975 | Rocket Pharmaceuticals Inc. | — |
| Recruiting | Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a NCT04784052 | Porteus, Matthew, MD | Phase 1 / Phase 2 |
| Completed | Eltrombopag for Treatment of Fanconi Anemia NCT06045052 | Julian Sevilla | Phase 2 |
| Active Not Recruiting | Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy NCT04437771 | Rocket Pharmaceuticals Inc. | — |
| Recruiting | Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Ma NCT04232085 | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Phase 2 |
| Recruiting | Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders NCT03579875 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Active Not Recruiting | Eltrombopag for People With Fanconi Anemia NCT03206086 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Terminated | A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia NCT03600909 | Memorial Sloan Kettering Cancer Center | Phase 2 |
| Active Not Recruiting | Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia NCT03476330 | Children's Hospital Medical Center, Cincinnati | Phase 2 |
| Completed | Pilot Study of Metformin for Patients With Fanconi Anemia NCT03398824 | Boston Children's Hospital | Phase 2 |
| Unknown | FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vect NCT03351868 | Shenzhen Geno-Immune Medical Institute | N/A |
| Completed | Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients NCT03609840 | University of California, San Francisco | — |
| Recruiting | Familial Investigations of Childhood Cancer Predisposition NCT03050268 | St. Jude Children's Research Hospital | — |
| Completed | Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Ple NCT02678533 | Assistance Publique - Hôpitaux de Paris | Phase 1 / Phase 2 |
| Terminated | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study NCT03733249 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Recruiting | Investigation of the Genetics of Hematologic Diseases NCT02720679 | St. Jude Children's Research Hospital | — |
| Completed | Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A NCT03157804 | Hospital Infantil Universitario Niño Jesús, Madrid, Spain | Phase 1 / Phase 2 |
| Terminated | Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant NCT02065869 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Recruiting | HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy NCT02143830 | Children's Hospital Medical Center, Cincinnati | Phase 2 |
| Completed | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases NCT01917708 | Emory University | Phase 1 |
| Completed | Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia fo NCT02931071 | Hospital Universitari Vall d'Hebron Research Institute | Phase 2 |
| Completed | Quercetin in Children With Fanconi Anemia; a Pilot Study NCT01720147 | Children's Hospital Medical Center, Cincinnati | Phase 1 |
| Terminated | Gene Therapy for Fanconi Anemia NCT01331018 | Fred Hutchinson Cancer Center | Phase 1 |
| Withdrawn | Unrelated HSCT in Patients With Fanconi Anemia NCT02127905 | Neena Kapoor, M.D. | N/A |
| Completed | Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation NCT01316549 | University of California, San Francisco | — |
| Terminated | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation NCT01319851 | Emory University | N/A |
| Terminated | Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita NCT01001598 | Boston Children's Hospital | Phase 1 / Phase 2 |
| Completed | Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia NCT01146210 | Children's Oncology Group | — |
| Completed | Hematopoietic Stem Cell Transplant for Fanconi Anemia NCT01071239 | Medical College of Wisconsin | Phase 2 |
| Completed | Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With NCT00856388 | Roswell Park Cancer Institute | N/A |
| Active Not Recruiting | Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at R NCT00542373 | M.D. Anderson Cancer Center | N/A |
| Completed | Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD NCT00479115 | Children's Hospital Medical Center, Cincinnati | Phase 1 / Phase 2 |
| Completed | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, NCT00453388 | Fred Hutchinson Cancer Center | Phase 2 |
| Completed | Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment NCT00499070 | University Hospital Freiburg | — |
| Completed | TBI Dose De-escalation for Fanconi Anemia NCT00352976 | Masonic Cancer Center, University of Minnesota | Phase 2 / Phase 3 |
| Completed | Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia NCT00965666 | Children's Hospital Medical Center, Cincinnati | EARLY_Phase 1 |
| Completed | Tissue Sample Collection From Patients With Fanconi Anemia NCT00899522 | OHSU Knight Cancer Institute | — |
| Completed | Blood Cell Collection for Future Use in Individuals With Fanconi Anemia NCT00271089 | National Heart, Lung, and Blood Institute (NHLBI) | N/A |
| Completed | Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia NCT00272857 | Boston Children's Hospital | Phase 1 |
| Completed | Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia NCT00243399 | Children's Hospital Medical Center, Cincinnati | Phase 1 |
| Terminated | Stem Cell Transplantation for Fanconi Anemia NCT00167206 | Masonic Cancer Center, University of Minnesota | Phase 1 / Phase 2 |
| Unknown | Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant D NCT00084695 | Milton S. Hershey Medical Center | Phase 2 |
| Completed | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Dis NCT01019876 | Columbia University | Phase 2 / Phase 3 |
| Completed | Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia NCT00258427 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Terminated | Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT NCT00586274 | Baylor College of Medicine | Phase 1 |
| Terminated | Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants NCT00896740 | OHSU Knight Cancer Institute | — |
| Completed | NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders NCT00053989 | Roswell Park Cancer Institute | Phase 2 |
| Recruiting | Cancer in Inherited Bone Marrow Failure Syndromes NCT00027274 | National Cancer Institute (NCI) | — |
| Terminated | Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia NCT00590460 | Baylor College of Medicine | Phase 1 / Phase 2 |
| Completed | Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically NCT00595127 | Memorial Sloan Kettering Cancer Center | N/A |
| Completed | Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young P NCT00305708 | University of California, San Francisco | Phase 1 / Phase 2 |
| Completed | Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi NCT00630253 | Masonic Cancer Center, University of Minnesota | Phase 1 / Phase 2 |
| Completed | Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in NCT00093743 | Fred Hutchinson Cancer Center | Phase 1 |
| Terminated | Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer NCT00290628 | Masonic Cancer Center, University of Minnesota | N/A |
| Completed | Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia NCT00317876 | Fred Hutchinson Cancer Center | Phase 1 |
| Completed | Cord Blood Stem Cell Transplantation Study (COBLT) NCT00000603 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma NCT00900055 | OHSU Knight Cancer Institute | — |
| Available | Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blo NCT06731036 | University of Florida | — |
| Available | Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children NCT01995305 | Xiaofan Zhu | — |