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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Bone Marrow Failure Disorders Clear all
Active, not recruiting Phase 1Phase 2 Interventional

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

NCT03301168
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic
Interventions
BPX-501 T cells, Rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
1 Month to 26 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2034
U.S. locations
10
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2022 · Synced Sep 22, 2026, 10:29 PM EDT
Completed Not applicable Interventional

Unrelated Donor Stem Cell Transplantation

NCT01364363
View directory record Official record
Conditions
Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Myeloproliferative Syndromes, Chronic Myelogenous Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Multiple Myeloma, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Large Granulocytic Leukemia
Interventions
Allogeneic transplantation
Procedure
Lead sponsor
Scripps Health
Other
Eligibility
18 Years to 69 Years
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2015
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Nov 22, 2022 · Synced Sep 22, 2026, 10:29 PM EDT
Completed Phase 2 Interventional

Combination Therapy of Severe Aplastic Anemia

NCT00001964
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Cyclosporine A, ATG, MMF
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Year to 99 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 15, 2021 · Synced Sep 22, 2026, 10:29 PM EDT
Active, not recruiting Not applicable Interventional Results available

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

NCT05236764
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Hemophagocytic Lymphohistiocytoses, Primary Immunodeficiency Diseases, Hemoglobinopathies, Severe Aplastic Anemia, Cytopenia, Bone Marrow Failure Syndrome, Severe Chronic Active Epstein-Barr Virus Infection
Interventions
CliniMACS
Device
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Sep 22, 2026, 10:29 PM EDT
Terminated Phase 2 Interventional

Unrelated Donor Transplant for Malignant and Non-Malignant Disorders

NCT01050439
View directory record Official record
Conditions
Leukemia, Lymphoma, Bone Marrow Failure Syndromes, Immunodeficiencies, Histiocytosis
Interventions
UDAlloSCT, Therapy
Procedure · Other
Lead sponsor
Columbia University
Other
Eligibility
Up to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 14, 2015 · Synced Sep 22, 2026, 10:29 PM EDT
Completed Not applicable Interventional Results available

Unrelated Umbilical Cord Blood (UBC)Transplantation

NCT01768845
View directory record Official record
Conditions
Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome, Multiple Myeloma, Hodgkin Lymphoma, Non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia (CLL), Acute Lymphocytic Leukemia (ALL), Severe Aplastic Anemia
Interventions
umbilical cord blood (UCB)
Genetic
Lead sponsor
West Virginia University
Other
Eligibility
16 Years to 70 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2021
U.S. locations
1
States / cities
Morgantown, West Virginia
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Sep 22, 2026, 10:29 PM EDT
Recruiting Phase 1Phase 2 Interventional

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT03128996
View directory record Official record
Conditions
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders, Hemoglobinopathies, Non-malignant Disorders
Interventions
RIC regimen, GVHD prophylaxis regimen
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2033
U.S. locations
4
States / cities
New Haven, Connecticut • Wilmington, Delaware • Grand Rapids, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Sep 22, 2026, 10:29 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia

NCT01193283
View directory record Official record
Conditions
Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
Interventions
Cyclophosphamide, Cyclosporine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 21, 2017 · Synced Sep 22, 2026, 10:29 PM EDT
Completed Phase 2 Interventional Results available

Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis

NCT02065154
View directory record Official record
Conditions
Leukemia, Lymphoma, Myelodysplastic Syndrome, Myelofibrosis, Severe Aplastic Anemia, Allogeneic Transplant
Interventions
Cyclophosphamide
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Sep 29, 2022 · Synced Sep 22, 2026, 10:29 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Renal Allograft Tolerance Through Mixed Chimerism

NCT01780454
View directory record Official record
Conditions
End Stage Renal Disease
Interventions
MEDI-507, Rituximab, Total Body Irradiation, Thymic Irradiation
Drug · Radiation
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 18, 2021 · Synced Sep 22, 2026, 10:29 PM EDT
Completed No phase listed Observational

Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy

NCT04436367
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Sep 22, 2026, 10:29 PM 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 22, 2026, 10:29 PM EDT
Completed No phase listed Observational

Fertility Preservation in Women Who Will Have Gonadotoxic Therapy or Hematopoetic Stem Cell Transplantation, and in Women With Sickle Cell Disease

NCT02225145
View directory record Official record
Conditions
Hemoglobin, Stem Cell Transplant, Aplastic Anemia
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
7 Years to 45 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 31, 2018 · Synced Sep 22, 2026, 10:29 PM EDT
Recruiting Early Phase 1 Interventional Accepts healthy volunteers

Bone Marrow Derived Stem Cells Mobilization for Treatment of Abnormal Endometrium

NCT05343572
View directory record Official record
Conditions
Asherman Syndrome, Atrophic Endometrium, Recurrent Implantation Failure
Interventions
Plerixafor
Drug
Lead sponsor
Hugh Taylor
Other
Eligibility
18 Years to 40 Years · Female only
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Orange, Connecticut
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 22, 2026, 10:29 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Feasibility Study of BMAC Enhanced CABG

NCT01074099
View directory record Official record
Conditions
Congestive Heart Failure
Interventions
BMAC, CABG only
Biological · Procedure
Lead sponsor
Harvest Technologies
Industry
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
2
States / cities
Houston, Texas • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jan 17, 2016 · Synced Sep 22, 2026, 10:29 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

NCT05757310
View directory record Official record
Conditions
Recurrent Severe Aplastic Anemia, Refractory Severe Aplastic Anemia
Interventions
Anti-Thymocyte Globulin, Biospecimen Collection, Bone Marrow Aspirate, Bone Marrow Biopsy, Cyclophosphamide, Haploidentical Hematopoietic Cell Transplantation, Pentostatin, Questionnaire Administration
Biological · Procedure · Drug + 1 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
Up to 75 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jan 4, 2026 · Synced Sep 22, 2026, 10:29 PM EDT
Terminated Phase 2 Interventional Results available

Low-Dose Decitabine in Myelodysplastic Syndrome Post Azacytidine Failure

NCT00113321
View directory record Official record
Conditions
Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia
Interventions
Decitabine
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 6, 2012 · Synced Sep 22, 2026, 10:29 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 26, 2026 · Synced Sep 22, 2026, 10:29 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Danazol for Genetic Bone Marrow and Lung Disorders

NCT01441037
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Danazol
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 14, 2018 · Synced Sep 22, 2026, 10:29 PM EDT
Completed No phase listed Observational

Collection of Blood From Patients With Pancytopenia

NCT00001214
View directory record Official record
Conditions
Pancytopenia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1986 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 20, 2020 · Synced Sep 22, 2026, 10:29 PM 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 Sep 22, 2026, 10:29 PM EDT
Active, not recruiting Phase 2 Interventional

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

NCT05796570
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 22, 2026, 10:29 PM EDT
Recruiting Phase 1 Interventional

UAB 2419-CD34 Selection Using the Automated CliniMACS Prodigy

NCT06047886
View directory record Official record
Conditions
AML, ALL, Lymphoid Malignancies, Myelodysplastic Syndromes, CML, Primary Myelofibrosis
Interventions
Infusion of CD34 selected hematopoietic stem cells
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
4 Weeks to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Sep 22, 2026, 10:29 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

Influenza Vaccine in Preventing Flu in Patients Who Have Undergone Stem Cell Transplant and in Healthy Volunteers

NCT00952185
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm, Viral Infection
Interventions
cytology specimen collection procedure, fluorescent antibody technique, assessment of therapy complications
Other · Procedure
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jun 7, 2015 · Synced Sep 22, 2026, 10:29 PM EDT