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Showing 1–24 of 100 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Fanconi Anemia Clear all
Enrolling by invitation No phase listed Observational

Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102

NCT07527975
View directory record Official record
Conditions
Fanconi Anemia
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2038
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Sep 6, 2026, 6:50 AM EDT
Recruiting No phase listed Observational

Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease

NCT07459582
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I, Glycogen Storage Disease Type IB, Glycogen Storage Disease Xi
Interventions
Lactate Meter, Accu Chek
Other · Device
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
Up to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Sep 6, 2026, 6:50 AM EDT
Completed Phase 2 Interventional

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

NCT00061763
View directory record Official record
Conditions
Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
6
States / cities
Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2017 · Synced Sep 6, 2026, 6:50 AM EDT
Recruiting Phase 1 Interventional

Study to Assess Safety, Tolerability and Activity of DSB2455 in Participants With Advanced Malignancies

NCT06458712
View directory record Official record
Conditions
Advanced Cancers (Breast, Ovarian, mCRPC, Pancreatic Ductal Adenocarcinoma (PDAC), Brain Mets) With Specific Mutations (Homologous Recombination Deficiency)
Interventions
DSB2455
Drug
Lead sponsor
Duke Street Bio Ltd
Industry
Eligibility
18 Years and older
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
4
States / cities
Sacramento, California • New Haven, Connecticut • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 6, 2026, 6:50 AM EDT
Recruiting Phase 2 Interventional

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

NCT04232085
View directory record Official record
Conditions
Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita, Telomere Biology Disorder, Short Telomere Syndrome
Interventions
Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil
Drug · Radiation
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
4 Months to 50 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 27, 2025 · Synced Sep 6, 2026, 6:50 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 Sep 6, 2026, 6:50 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 Sep 6, 2026, 6:50 AM EDT
Active, not recruiting Phase 2 Interventional

Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant

NCT03924401
View directory record Official record
Conditions
Graft Versus Host Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 20 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Wilmington, Delaware • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2026 · Synced Sep 6, 2026, 6:50 AM EDT
Completed Phase 1 Interventional

Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

NCT00272857
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Retrovirus Construct
Genetic
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Year to 35 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 22, 2017 · Synced Sep 6, 2026, 6:50 AM EDT
Completed No phase listed Observational

Fanconi Syndrome Due to ARVs in HIV-Infected Persons

NCT00499187
View directory record Official record
Conditions
HIV Infections, Fanconi Syndrome, Kidney Disease, Renal Impairment
Interventions
Blood Draws
Procedure
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
10
States / cities
Los Angeles, California • Denver, Colorado • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2014 · Synced Sep 6, 2026, 6:50 AM EDT
Not listed Phase 1 Interventional

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

NCT03814408
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year to 12 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Sep 6, 2026, 6:50 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 Sep 6, 2026, 6:50 AM EDT
Recruiting Phase 2 Interventional

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

NCT02143830
View directory record Official record
Conditions
Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Interventions
Busulfan, Cyclophosphamide, Fludarabine, rabbit ATG, G-CSF, Peripheral blood stem cell
Drug · Biological
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Months and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2028
U.S. locations
3
States / cities
New York, New York • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 6, 2026, 6:50 AM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 6, 2026, 6:50 AM EDT
Completed Phase 2Phase 3 Interventional Results available

TBI Dose De-escalation for Fanconi Anemia

NCT00352976
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone Marrow Transplantation, Mycophenolate Mofetil, Sirolimus
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 23, 2021 · Synced Sep 6, 2026, 6:50 AM EDT
Completed Phase 2 Interventional Results available

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia

NCT00453388
View directory record Official record
Conditions
Acute Myeloid Leukemia in Remission, de Novo Myelodysplastic Syndrome, Fanconi Anemia, Previously Treated Myelodysplastic Syndrome
Interventions
Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
4
States / cities
Oakland, California • Nashville, Tennessee • Seattle, Washington + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2020 · Synced Sep 6, 2026, 6:50 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 Sep 6, 2026, 6:50 AM EDT
Completed Phase 2 Interventional

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

NCT01044186
View directory record Official record
Conditions
Transfusional Iron Overload
Interventions
ICL670
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
8
States / cities
Oakland, California • Orange, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2017 · Synced Sep 6, 2026, 6:50 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 Sep 6, 2026, 6:50 AM EDT
Recruiting No phase listed Observational

Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia

NCT06744283
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 6, 2026, 6:50 AM EDT
Recruiting No phase listed Observational

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

NCT07649031
View directory record Official record
Conditions
Fanconi Anemia
Interventions
MRIs
Diagnostic Test
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 6, 2026, 6:50 AM EDT
Completed Phase 2 Interventional Results available

Assessment of Efficacy of AZD2281 in Platinum Sensitive Relapsed Serous Ovarian Cancer

NCT00753545
View directory record Official record
Conditions
Ovarian Cancer
Interventions
AZD2281, matching placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years · Female only
Enrollment
265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2023
U.S. locations
11
States / cities
Berkeley, California • San Francisco, California • West Hollywood, California + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2025 · Synced Sep 6, 2026, 6:50 AM EDT
Not listed Phase 1 Interventional

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

NCT00006127
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
amifostine
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
2 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 6, 2026, 6:50 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 Sep 6, 2026, 6:50 AM EDT