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Showing 1–24 of 121 matching trials from the live ClinicalTrials.gov search.
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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
Conditions
Fanconi Anemia
Interventions
AMD3100, AmCell CliniMACs
Drug · Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Year to 30 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Cincinnati, Ohio
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
Conditions
Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions
Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Telomere Biology Disorders, Dyskeratosis Congenita, Revesz Syndrome, Hoyeraal Hreidarsson Syndrome, Telomere Biology Disorders With Bone Marrow Failure, Interstitial Lung Disease Due to Systemic Disease (Telomere Biology Disorder), Pulmonary Fibrosis, Familial (Telomere Biology Disorder)
Interventions
deoxycytidine, deoxythymidine
Drug
Lead sponsor
Suneet Agarwal
Other
Eligibility
1 Year to 70 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Boston, Massachusetts
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
Conditions
Fanconi Anemia
Interventions
JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant, Rabbit Anti-Thymoglobulin (rATG), Cyclophosphamide, Fludarabine, Rituximab
Drug · Device · Biological
Lead sponsor
Porteus, Matthew, MD
Other
Eligibility
2 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Stanford, California
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
Conditions
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Interventions
imatinib, TruSight Oncology
Drug · Device
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 120 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Fanconi Anemia
Interventions
anti-thymocyte globulin, filgrastim, busulfan, cyclophosphamide, fludarabine phosphate, methylprednisolone, Hematopoietic stem cell transplantation
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 44 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
Conditions
Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction, protein expression analysis, reverse transcriptase-polymerase chain reaction, western blotting, chromatography, high performance liquid chromatography, immunoenzyme technique
Genetic · Other
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
213 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1975 – 2016
U.S. locations
1
States / cities
Portland, Oregon
Conditions
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
Interventions
Treosulfan, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography
Drug · Biological · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 49 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
24
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 20 more
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
Conditions
Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic Anemia
Interventions
alemtuzumab, Fludarabine, Cyclosporins, Mycophenolate mofetil, Tacrolimus
Biological · Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
30 Days to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2034
U.S. locations
11
States / cities
Chicago, Illinois • Boston, Massachusetts • Kansas City, Missouri + 6 more
Terminated Phase 1Phase 2 Interventional Results available

Stem Cell Transplantation for Fanconi Anemia

NCT00167206
Conditions
Fanconi Anemia
Interventions
Hematopoietic Stem Cell Transplant, Thymic Shielding During Radiation, Total Body Irradiation, Cyclophosphamide, Fludarabine
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
1 Day to 18 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Fanconi Anemia, Leukemia, Severe Congenital Neutropenia, Thrombocytopenia
Interventions
anti-thymocyte globulin, busulfan, fludarabine phosphate, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2004
U.S. locations
1
States / cities
San Francisco, California
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA

NCT00455312
Conditions
Dyskeratosis Congenita, Aplastic Anemia
Interventions
Campath 1H, Cyclophosphamide, Fludarabine, Total Body Irradiation, Stem Cell Transplantation, antithymocyte globulin, Methylprednisolone
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 70 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
Conditions
Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloid Malignancies, MDS, Inherited Bone Marrow Failure Syndrome, Myeloid Neoplasm, Aml
Interventions
Decitabine, Filgrastim
Drug
Lead sponsor
Franziska Wachter
Other
Eligibility
1 Year to 39 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
2
States / cities
Boston, Massachusetts
Conditions
Anemia, Aplastic, Red-Cell Aplasia, Pure, Anemia, Diamond-Blackfan
Interventions
Rituximab
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
Drug · Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2013
U.S. locations
1
States / cities
New York, New York