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Showing 1–24 of 111 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Fanconi Syndrome Clear all
Completed Phase 3 Interventional Results available

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

NCT01197378
View directory record Official record
Conditions
Cystinosis
Interventions
Cysteamine Bitartrate Delayed-release Capsules
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Year and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
5
States / cities
San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 26, 2024 · Synced Sep 10, 2026, 8:52 PM EDT
Completed No phase listed Observational

Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

NCT01316549
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 3, 2021 · Synced Sep 10, 2026, 8:52 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

NCT00872729
View directory record Official record
Conditions
Cystinosis
Interventions
Cystagon®, RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Sep 10, 2026, 8:52 PM EDT
Completed No phase listed Observational

The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study

NCT00071903
View directory record Official record
Conditions
Pseudotumor Cerebri, Cystinosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 50 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 10, 2026, 8:52 PM 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 Sep 10, 2026, 8:52 PM EDT
Terminated Phase 1 Interventional Results available

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

NCT02231710
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions
BPX-501 and Rimiducid
Biological
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
4 Months to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 24, 2024 · Synced Sep 10, 2026, 8:52 PM 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 Sep 10, 2026, 8:52 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants

NCT00896740
View directory record Official record
Conditions
Fanconi Anemia
Interventions
microarray analysis, biopsy
Genetic · Procedure
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2007
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 3, 2017 · Synced Sep 10, 2026, 8:52 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT

NCT00586274
View directory record Official record
Conditions
FANCONI ANEMIA
Interventions
CD34 selected haploidentical PBSCT, Fludarabine, T cell infusion, Campath 1h, anti-CD45
Procedure · Drug · Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 64 Years
Enrollment
1 participant
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2009
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 3, 2014 · Synced Sep 10, 2026, 8:52 PM 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 Sep 10, 2026, 8:52 PM EDT
Completed Phase 2 Interventional

Single Arm Study of BSI-201 in Patients With BRCA-1 or BRCA-2 Associated Advanced Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer

NCT00677079
View directory record Official record
Conditions
Primary Peritoneal Cancer, Advanced Epithelial Ovarian Cancer
Interventions
Iniparib
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years and older · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 30, 2013 · Synced Sep 10, 2026, 8:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

DFT383 in Pediatric Participants With Nephropathic Cystinosis

NCT06910813
View directory record Official record
Conditions
Nephropathic Cystinosis
Interventions
DFT383
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 5 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2044
U.S. locations
5
States / cities
Phoenix, Arizona • San Diego, California • Stanford, California + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Sep 10, 2026, 8:52 PM EDT
Active, not recruiting Phase 2 Interventional

Eltrombopag for People With Fanconi Anemia

NCT03206086
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
6 Years to 99 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 23, 2025 · Synced Sep 10, 2026, 8:52 PM 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 10, 2026, 8:52 PM 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 Sep 10, 2026, 8:52 PM 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 10, 2026, 8:52 PM EDT
Terminated Not applicable Interventional

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

NCT00290628
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, filgrastim, melphalan, methylprednisolone, mycophenolate mofetil, radiation therapy, umbilical cord blood transplantation
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 45 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2007
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 28, 2017 · Synced Sep 10, 2026, 8:52 PM EDT
Not yet recruiting Phase 2 Interventional

Vilastobart+Retifanlimab in BRCA or PALB2 Deficient PC

NCT07765836
View directory record Official record
Conditions
Pancreatic Cancer Metastatic
Interventions
vilastobart (XTX101), Retifanlimab
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Sep 10, 2026, 8:52 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluating Cell Damage in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, or Fanconi Anemia; in Patients Who Were Exposed to Alkylating Agents; and in Healthy Volunteers

NCT00899795
View directory record Official record
Conditions
Leukemia, Myelodysplastic Syndromes
Interventions
cytogenetic analysis, fluorescence in situ hybridization, flow cytometry, biopsy
Genetic · Other · Procedure
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
5 Years to 120 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2010
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 3, 2017 · Synced Sep 10, 2026, 8:52 PM 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 Sep 10, 2026, 8:52 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Lynne Cohen Consortium Project: Multi-Center Database/Registry

NCT00776958
View directory record Official record
Conditions
Ovarian Cancer, Breast Cancer
Interventions
Questionnaire
Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older · Female only
Enrollment
176 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2008
U.S. locations
4
States / cities
Birmingham, Alabama • Los Angeles, California • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2014 · Synced Sep 10, 2026, 8:52 PM 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 Sep 10, 2026, 8:52 PM EDT
Recruiting No phase listed Observational

Familial Investigations of Childhood Cancer Predisposition

NCT03050268
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Sep 10, 2026, 8:52 PM 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 10, 2026, 8:52 PM EDT