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ClinicalTrials.gov public records Last synced Oct 3, 2026, 11:24 PM EDT

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Showing 25–48 of 108 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Hypoplastic Anemia Clear all
Terminated Phase 2 Interventional Results available

A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

NCT00001962
View directory record Official record
Conditions
Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia
Interventions
Daclizumab
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2021 · Synced Oct 3, 2026, 11:24 PM EDT
Active, not 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
2024 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100

NCT00479115
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 30, 2020 · Synced Oct 3, 2026, 11:24 PM 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 4, 2008 · Synced Oct 3, 2026, 11:24 PM 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 24, 2005 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 2 Interventional Results available

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

NCT00919503
View directory record Official record
Conditions
Non-Neoplastic Hematologic and Lymphocytic Disorder
Interventions
Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
Procedure · Biological · Drug + 2 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 49 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
6
States / cities
Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2021 · Synced Oct 3, 2026, 11:24 PM 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 28, 2025 · Synced Oct 3, 2026, 11:24 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Multi-Center Study of Iron Overload: Pilot Study

NCT01114776
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions
Not listed
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Sep 24, 2020 · Synced Oct 3, 2026, 11:24 PM EDT
Recruiting Phase 1Phase 2 Interventional

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

NCT04784052
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 13, 2014 · Synced Oct 3, 2026, 11:24 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 24, 2021 · Synced Oct 3, 2026, 11:24 PM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 2 Interventional

Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

NCT00011505
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
G-CSF, Leukapheresis
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 3, 2026, 11:24 PM EDT
Completed No phase listed Observational

Tissue Sample Collection From Patients With Fanconi Anemia

NCT00899522
View directory record Official record
Conditions
Fanconi Anemia, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
biologic sample preservation procedure, biopsy
Other · Procedure
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced Oct 3, 2026, 11:24 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 24, 2018 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 2 Interventional Results available

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

NCT00053989
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Fanconi Anemia, Aplastic Anemia
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim, therapeutic allogeneic lymphocytes, cyclophosphamide, fludarabine phosphate, methylprednisolone, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years to 75 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2018
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Feb 10, 2020 · Synced Oct 3, 2026, 11:24 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 25, 2024 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 1Phase 2 Interventional

Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

NCT00305708
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 12, 2012 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 1 Interventional

Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia

NCT00317876
View directory record Official record
Conditions
Fanconi Anemia
Interventions
cyclophosphamide, cyclosporine, methotrexate, allogeneic bone marrow transplantation, nonmyeloablative allogeneic hematopoietic stem cell transplantation
Drug · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
2
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 20, 2012 · Synced Oct 3, 2026, 11:24 PM EDT
Completed Phase 2 Interventional Results available

Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

NCT00957931
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions
Bone marrow transplantation, Mesenchymal Stromal Cells
Procedure · Biological
Lead sponsor
Stanford University
Other
Eligibility
1 Year to 25 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Birmingham, Alabama • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 6, 2018 · Synced Oct 3, 2026, 11:24 PM EDT
Terminated Not applicable Interventional Results available

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

NCT01319851
View directory record Official record
Conditions
Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic-granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Schwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Fanconi Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia
Interventions
Alefacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 27, 2017 · Synced Oct 3, 2026, 11:24 PM 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 14, 2020 · Synced Oct 3, 2026, 11:24 PM EDT
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 3, 2026, 11:24 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 3, 2023 · Synced Oct 3, 2026, 11:24 PM EDT