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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Anemia, Diamond-Blackfan Clear all
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 12, 2021 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Phase 2 Interventional

Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis

NCT00578435
View directory record Official record
Conditions
Genetic Disorders, Sickle Cell Anemia
Interventions
Busulfan, Cyclophosphamide, BMD
Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2008
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 11, 2008 · Synced Jun 10, 2026, 9:46 AM EDT
Terminated Phase 1 Interventional Results available

Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

NCT01034592
View directory record Official record
Conditions
Anemia, Leukemia, Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)
Interventions
Lenalidomide
Drug
Lead sponsor
Jason Robert Gotlib
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Aug 1, 2017 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

NCT00005893
View directory record Official record
Conditions
Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
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 Jun 10, 2026, 9:46 AM 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 5, 2018 · Synced Jun 10, 2026, 9:46 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

NCT01966367
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions
CD34 Stem Cell Selection Therapy
Biological
Lead sponsor
Diane George
Other
Eligibility
Up to 40 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 13, 2025 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Jun 10, 2026, 9:46 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

The Use of Trifluoperazine in Transfusion Dependent DBA

NCT03966053
View directory record Official record
Conditions
Diamond Blackfan Anemia, Pure Red Cell Aplasia
Interventions
Trifluoperazine
Drug
Lead sponsor
Adrianna Vlachos, MD
Other
Eligibility
18 Years to 65 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Boston, Massachusetts • Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Dec 13, 2022 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Not applicable Interventional Results available

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

NCT00244010
View directory record Official record
Conditions
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions
Allogeneic stem cell transplant
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Jun 10, 2026, 9:46 AM 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 11, 2012 · Synced Jun 10, 2026, 9:46 AM 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 3, 2008 · Synced Jun 10, 2026, 9:46 AM 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 26, 2017 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Phase 3 Interventional Results available

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

NCT00235391
View directory record Official record
Conditions
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
1,683 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
27
States / cities
Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 6, 2011 · Synced Jun 10, 2026, 9:46 AM EDT
Terminated Phase 2 Interventional Results available

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

NCT03333486
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
Drug · Other · Procedure + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
1 Year to 75 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced Jun 10, 2026, 9:46 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 Jun 10, 2026, 9:46 AM EDT
Recruiting No phase listed Observational

Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)

NCT07186179
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
Mobilization Regimen
Drug
Lead sponsor
Northwell Health
Other
Eligibility
3 Years to 30 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
New Hyde Park, New York
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Jun 10, 2026, 9:46 AM EDT
Recruiting Phase 1Phase 2 Interventional

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

NCT03653338
View directory record Official record
Conditions
Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
Interventions
CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa
Biological · Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 11, 2025 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Phase 1Phase 2 Interventional

Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia

NCT05828108
View directory record Official record
Conditions
Steroid-refractory Diamond-Blackfan Anemia (DBA)
Interventions
Bitopertin
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Jun 10, 2026, 9:46 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 Jun 10, 2026, 9:46 AM EDT
Completed Phase 2 Interventional Results available

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

NCT00301834
View directory record Official record
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions
alemtuzumab, busulfan, cyclosporine, fludarabine phosphate, methotrexate, methylprednisolone, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 21 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
2
States / cities
San Francisco, California • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 27, 2017 · Synced Jun 10, 2026, 9:46 AM EDT
Completed No phase listed Observational

Multi-Center Study of Iron Overload: Survey Study (MCSIO)

NCT01913548
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
423 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
6
States / cities
Oakland, California • Augusta, Georgia • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2020 · Synced Jun 10, 2026, 9:46 AM 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 2, 2023 · Synced Jun 10, 2026, 9:46 AM EDT
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Jun 10, 2026, 9:46 AM EDT