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Showing 1–24 of 267 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Bone Marrow Aplasia Clear all
Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita

NCT01001598
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 18, 2019 · Synced Sep 16, 2026, 9:10 AM 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 29, 2020 · Synced Sep 16, 2026, 9:10 AM EDT
Not listed Phase 1Phase 2 Interventional

Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

NCT00319878
View directory record Official record
Conditions
Anemia, Aplastic
Interventions
Sirolimus, Cyclosporine
Drug
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
21 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
4
States / cities
Los Angeles, California • Tampa, Florida • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2008 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Not applicable Interventional Results available

Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies

NCT01499147
View directory record Official record
Conditions
Acute Myeloid Leukemia, Acute Leukemia, Chronic Myelogenous Leukemia, Malignant Lymphoma, Hodgkin's Disease, Multiple Myeloma, Lymphocytic Leukemia, Myeloproliferative Disorder, Polycythemia Vera, Myelofibrosis, Aplastic Anemia
Interventions
fludarabine/busulfan, fludarabine/ melphalan, ATG
Drug
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
10 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2013
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 7, 2018 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 4 Interventional

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

NCT03176849
View directory record Official record
Conditions
Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder, Pediatric Acute Myeloid Leukemia, Pediatric Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Sickle Cell Anemia in Children, Aplastic Anemia, Thalassemia in Children
Interventions
Vitamin D3, Standard Vitamin D3 Supplementation
Dietary Supplement
Lead sponsor
Phoenix Children's Hospital
Other
Eligibility
1 Year to 25 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 16, 2020 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 1Phase 2 Interventional

Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation of Patients With Hematological Diseases

NCT00056966
View directory record Official record
Conditions
Hematologic Malignancy
Interventions
ANTI-CD45, CAMPATH-1H, FK506, Fludarabine, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 28, 2012 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 2 Interventional Results available

Extended Dosing With Eltrombopag for Severe Aplastic Anemia

NCT01891994
View directory record Official record
Conditions
Severe Aplastic Anemia (SAA)
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 24, 2023 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 26, 2020 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Not applicable Interventional

Nonmyeloablative Allogeneic Transplant

NCT01272817
View directory record Official record
Conditions
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Mantle Cell Lymphoma, Multiple Myeloma, Waldenstrom Macroglobulinemia, Breast Cancer, Renal Cell Carcinoma, Melanoma, Sarcoma, Ovarian Cancer, Thymoma
Interventions
Nonmyeloablative Allogeneic Transplant
Procedure
Lead sponsor
Scripps Health
Other
Eligibility
18 Years to 72 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2015
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Nov 22, 2022 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 1 Interventional

Safety and Efficacy of Obatoclax Mesylate (GX15-070MS) for the Treatment of Hematological Malignancies

NCT00438178
View directory record Official record
Conditions
Hematological Malignancies
Interventions
Obatoclax mesylate (GX15-070MS)
Drug
Lead sponsor
Gemin X
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 11, 2014 · Synced Sep 16, 2026, 9:10 AM EDT
Recruiting Phase 2 Interventional

Haploidentical HCT for Severe Aplastic Anemia

NCT04558736
View directory record Official record
Conditions
Aplastic Anemia, Bone Marrow Failure Syndrome
Interventions
Anti-Thymocyte Globulin (Rabbit), Fludarabine, Cyclophosphamide, Mesna, G-CSF, Total Lymphoid Irradiation (TLI), CliniMACS, HPC, A Infusion, CD45RA-depleted DLI
Drug · Radiation · Device + 1 more
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 2 Interventional

Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia

NCT00004323
View directory record Official record
Conditions
Aplastic Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, mesna, methotrexate, methylprednisolone
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 55 Years
Enrollment
10 participants
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 Sep 16, 2026, 9:10 AM 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 16, 2026, 9:10 AM EDT
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 16, 2026, 9:10 AM 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 16, 2026, 9:10 AM 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 27, 2025 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 2 Interventional

Comparing Therapies for the Treatment of Severe Aplastic Anemia

NCT00001626
View directory record Official record
Conditions
Severe Aplastic Anemia (SAA)
Interventions
antithymocyte globulin, cyclophosphamide, cyclosporine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
15 Years to 110 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 21, 2020 · Synced Sep 16, 2026, 9:10 AM EDT
Recruiting Phase 2 Interventional

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

NCT06412497
View directory record Official record
Conditions
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
Interventions
Rituximab, Rabbit ATG, Cyclophosphamide, Fludarabine, Total Body Irradiation, Cell Infusion, Post-Transplant G-CSF, Tacrolimus, Mycophenolate Mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2036
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Sep 16, 2026, 9:10 AM EDT
Completed No phase listed Observational

Investigation of the Cylex® ImmuKnow® Assay

NCT00569842
View directory record Official record
Conditions
Leukemia, Non-Hodgkin's Lymphoma, Chronic Lymphocytic Leukemia, Hodgkin's Disease, Multiple Myeloma, Myelodysplastic Syndromes, Myeloproliferative Disorders, Aplastic Anemia, Chronic Myelogenous Leukemia
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Sep 10, 2014 · Synced Sep 16, 2026, 9:10 AM EDT
Terminated Phase 2 Interventional

Thrombosomes® in Bleeding Thrombocytopenic Patients Study

NCT04631211
View directory record Official record
Conditions
Thrombocytopenia, Hematologic Malignancy, Bone Marrow Aplasia, Myeloproliferative Disorders, Myelodysplastic Syndromes, Platelet Refractoriness
Interventions
Thrombosomes, Liquid Stored Platelets (LSP)
Biological
Lead sponsor
Cellphire Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
4
States / cities
Duarte, California • Washington D.C., District of Columbia • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated May 24, 2023 · Synced Sep 16, 2026, 9:10 AM EDT
Recruiting Phase 2 Interventional

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

NCT06517641
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Haploidentical donor bone marrow transplant, Unrelated donor bone marrow transplant
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
3 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
25
States / cities
Birmingham, Alabama • Duarte, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Sep 16, 2026, 9:10 AM EDT
Recruiting Phase 1Phase 2 Interventional

Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

NCT06303193
View directory record Official record
Conditions
Myelodysplastic Syndromes
Interventions
pacritinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 120 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Phase 2 Interventional Results available

Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

NCT00258427
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 1, 2021 · Synced Sep 16, 2026, 9:10 AM EDT
Completed Not applicable Interventional

Sharing Our Strength: A Research Study for Bone Marrow/Stem Cell Transplant Survivors

NCT00701844
View directory record Official record
Conditions
Leukemia, Acute Leukemia, Acute Lymphocytic Leukemia, Multiple Myeloma, Hematological Cancers, Psychological Distress
Interventions
Writing A (Experimental informative writing), Writing B (Experimental noninformative writing), Writing C (Control informative writing), Writing D (Control noninformative writing)
Behavioral
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years and older
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
3
States / cities
Hackensack, New Jersey • New York, New York • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 16, 2017 · Synced Sep 16, 2026, 9:10 AM EDT