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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: FANCONI ANEMIA Clear all
Completed Phase 1 Interventional

Gene Therapy for the Treatment of Fanconi's Anemia Type C

NCT00001399
View directory record Official record
Conditions
Fanconi's Anemia, Pancytopenia
Interventions
Transduced CD34+ Cells
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
5 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia

NCT00005898
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, filgrastim, fludarabine, methylprednisolone
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
0 Years to 34 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 24, 2026, 12:58 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations

NCT04042831
View directory record Official record
Conditions
Bile Duct Adenocarcinoma, Fanconi Anemia Complementation Group Gene Mutation, Metastatic Bile Duct Carcinoma, PTEN Gene Deletion
Interventions
Biospecimen Collection, Computed Tomography, Magnetic Resonance Imaging, Olaparib
Procedure · Drug
Lead sponsor
Academic and Community Cancer Research United
Other
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Early Phase 1 Interventional

Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

NCT00965666
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Etanercept
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
4 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 13, 2020 · Synced Jul 24, 2026, 12:58 AM EDT
Recruiting No phase listed Observational

Investigation of the Genetics of Hematologic Diseases

NCT02720679
View directory record Official record
Conditions
Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis, Blood Coagulation Disorder, Sickle Cell Disease, Dyskeratosis Congenita, Diamond-Blackfan Anemia, Congenital Thrombocytopenia, Severe Congenital Neutropenia, Fanconi Anemia, Myelodysplastic Syndromes, Myeloproliferative Diseases
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,716 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2050
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Phase 2Phase 3 Interventional

Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases

NCT01019876
View directory record Official record
Conditions
Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
Interventions
Fludarabine, Cyclophosphamide, Busulfan, Alemtuzumab, Rabbit Anti-thymocyte Globulin, Horse Anti-thymocyte Globulin
Drug
Lead sponsor
Columbia University
Other
Eligibility
Up to 30 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 30, 2024 · Synced Jul 24, 2026, 12:58 AM EDT
Terminated Phase 1 Interventional

Gene Therapy for Fanconi Anemia

NCT01331018
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells, Laboratory Biomarker Analysis, Leukapheresis, Methylprednisolone, Plerixafor, Prednisone
Procedure · Biological · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
4 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2024
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Jul 24, 2026, 12:58 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

FaCT Trial (Facilitated Cascade Testing Trial)

NCT04613440
View directory record Official record
Conditions
BRCA1 Mutation, BRCA2 Mutation
Interventions
Family Letter (Standard of Care), Educational Video, Mailed Saliva Kit for Genetic Testing, Telephone Counseling
Other
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older
Enrollment
524 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
3
States / cities
New York, New York • Durham, North Carolina • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 24, 2026, 12:58 AM EDT
Active, not recruiting Phase 2 Interventional

Gene Therapy for Fanconi Anemia, Complementation Group A

NCT04248439
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
2
States / cities
Stanford, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Phase 1 Interventional Results available

Study of Talazoparib, a PARP Inhibitor, in Patients With Advanced or Recurrent Solid Tumors

NCT01286987
View directory record Official record
Conditions
Advanced or Recurrent Solid Tumors, Breast Neoplasms, Ovarian Cancer, Epithelial, Ewing Sarcoma, Small Cell Lung Carcinoma, Prostate Cancer, Pancreas Cancer
Interventions
Talazoparib
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
14
States / cities
Scottsdale, Arizona • Los Angeles, California • Santa Monica, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jan 9, 2019 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Phase 2 Interventional Results available

Pilot Study of Metformin for Patients With Fanconi Anemia

NCT03398824
View directory record Official record
Conditions
Fanconi Anemia
Interventions
metformin HCl
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years to 35 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 28, 2022 · Synced Jul 24, 2026, 12:58 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita

NCT01001598
View directory record Official record
Conditions
Fanconi Anemia, Dyskeratosis Congenita
Interventions
danazol
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 18, 2019 · Synced Jul 24, 2026, 12:58 AM EDT
Completed No phase listed Observational

A Qualitative Exploration of the Impact of Positive BRCA1/2 Mutation Status on the Lives of Young Women

NCT00858078
View directory record Official record
Conditions
BRCA1 Gene, BRCA2 Gene
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 100 Years · Female only
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 29, 2021 · Synced Jul 24, 2026, 12:58 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Role of Acetaldehyde in the Development of Oral Cancer

NCT05973656
View directory record Official record
Conditions
Alcohol-Related Carcinoma, Fanconi Anemia, Oral Cavity Carcinoma
Interventions
Alcohol, Biospecimen Collection, Breath Test
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years to 45 Years
Enrollment
170 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Jul 24, 2026, 12:58 AM EDT
Terminated No phase listed Observational

Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia

NCT05910853
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Specimen Donation
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 85 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced Jul 24, 2026, 12:58 AM EDT
Not listed Phase 2 Interventional

Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases

NCT00084695
View directory record Official record
Conditions
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, fludarabine phosphate, melphalan, methylprednisolone, radiation therapy
Biological · Drug · Radiation
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 9, 2014 · Synced Jul 24, 2026, 12:58 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Cancer of the Pancreas Screening Study (CAPS 3)

NCT00438906
View directory record Official record
Conditions
Pancreatic Neoplasm, Peutz-Jeghers Syndrome
Interventions
Biopsy, Fine Needle Aspiration (FNA), Secretin (human synthetic) - ChiRhoClin
Procedure · Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
40 Years to 80 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2009
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia

NCT00005892
View directory record Official record
Conditions
Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
Interventions
cyclophosphamide, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 54 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 24, 2026, 12:58 AM EDT
Withdrawn No phase listed Observational

Clinical Outcomes in Hereditary Cancer

NCT00580021
View directory record Official record
Conditions
Breast Cancer, Pancreatic Cancer
Interventions
Genotyping
Other
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 27, 2016 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Phase 2 Interventional

Medical Treatment for Diamond Blackfan Anemia

NCT00001749
View directory record Official record
Conditions
Fanconi's Anemia, Hematologic Disease
Interventions
Antithymocyte globulin, Cyclosporine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Not applicable Interventional

Counseling Interventions for BRCA 1/2 Cancer Susceptibility Testing

NCT00165152
View directory record Official record
Conditions
Breast Cancer, Ovarian Cancer
Interventions
Genetic Counseling, Informed Consent Counseling
Procedure
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older · Female only
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2005
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 8, 2012 · Synced Jul 24, 2026, 12:58 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

NCT00987480
View directory record Official record
Conditions
Aplastic Anemia, Leukemia, Myelodysplastic Syndrome
Interventions
Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine., CliniMACS device
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
6
States / cities
Boston, Massachusetts • New York, New York • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2018 · Synced Jul 24, 2026, 12:58 AM EDT
Not listed No phase listed Observational

Cure Cystinosis International Registry

NCT01327807
View directory record Official record
Conditions
Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
Interventions
Not listed
Lead sponsor
Cystinosis Research Foundation
Other
Eligibility
Not listed
Enrollment
750 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2022
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 11, 2014 · Synced Jul 24, 2026, 12:58 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia

NCT00590460
View directory record Official record
Conditions
Fanconi Anemia, Severe Aplastic Anemia
Interventions
CAMPATH-1H, Anti-CD45, Fludarabine, Stem cell infusion
Biological · Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 23, 2018 · Synced Jul 24, 2026, 12:58 AM EDT