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Showing 1–14 of 14 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Acquired Bone Marrow Failure Syndromes Clear all
Recruiting No phase listed Observational

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

NCT05012111
View directory record Official record
Conditions
Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2041
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Jul 21, 2026, 11:48 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 Jul 21, 2026, 11:48 AM EDT
Recruiting Phase 3 Interventional

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

NCT06056297
View directory record Official record
Conditions
Neutropenia
Interventions
Mavorixafor, Placebo
Drug
Lead sponsor
X4 Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
22
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Jul 21, 2026, 11:48 AM EDT
Active, not recruiting Phase 2 Interventional

Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

NCT06752694
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Bone Marrow Transplantation, Computed Tomography, Cyclosporine, Fludarabine, Granulocyte Colony-Stimulating Factor, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Ruxolitinib, Sirolimus, Total-Body Irradiation, Multigated Acquisition Scan, Echocardiography
Procedure · Drug · Radiation
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Jul 21, 2026, 11:48 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 3, 2024 · Synced Jul 21, 2026, 11:48 AM EDT
Active, not recruiting Not applicable Interventional

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

NCT03047746
View directory record Official record
Conditions
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
Interventions
CliniMACs
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 25 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 21, 2026, 11:48 AM EDT
Completed Phase 1 Interventional

Stem Cell Factor Medication for Aplastic Anemia

NCT00001398
View directory record Official record
Conditions
Aplastic Anemia, Pancytopenia
Interventions
Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 21, 2026, 11:48 AM EDT
Recruiting Phase 2 Interventional

Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

NCT03314974
View directory record Official record
Conditions
Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Lymphoma, Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myeloproliferative Neoplasms, Myelofibrosis, Myelodysplasia, Refractory Anemia, High Risk Anemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Diffuse Large Cell Non Hodgkins Lymphoma, Lymphoblastic Lymphoma, Burkitt Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult, Multiple Myeloma, Juvenile Myelomonocytic Leukemia, Biphenotypic/Undifferentiated/Prolymphocytic Leukemias, MRD Positive Leukemia, Natural Killer Cell Malignancies, Acquired Bone Marrow Failure Syndromes
Interventions
HSCT with TBI Regimen, HSCT with Non-TBI Regimen
Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 60 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 21, 2026, 11:48 AM EDT
Active, not recruiting Early Phase 1 Interventional

Fludarabine Based RIC for Bone Marrow Failure Syndromes

NCT02928991
View directory record Official record
Conditions
Bone Marrow Failure Syndromes
Interventions
MRD-BMT with Fludarabine-based RIC for Acquired AA, MRD-BMT with Fludarabine-based RIC for iBMF with trilineage aplasia, MRD-BMT with Fludarabine-based RIC for iBMF without trilineage aplasia
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 22 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Jul 21, 2026, 11:48 AM EDT
No Longer Available No phase listed Expanded access

Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

NCT01200017
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis, Cytopenias, Leukocyte Disorders, Anemia Due to Intrinsic Red Cell Abnormality
Interventions
CD34+ enriched, T Cell Depleted donor stem cell product
Biological
Lead sponsor
Christopher Dvorak
Other
Eligibility
2 Months to 30 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 26, 2021 · Synced Jul 21, 2026, 11:48 AM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

NCT06839456
View directory record Official record
Conditions
Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
Interventions
Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Month to 25 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Jul 21, 2026, 11:48 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 Jul 21, 2026, 11:48 AM EDT
Completed Early Phase 1 Interventional

TPO-Mimetic Use in Children for Hematopoietic Failure

NCT04478227
View directory record Official record
Conditions
Bone Marrow Failure Disorders, Aplastic Anemia, Thrombocytopenia, Refractory Cytopenia of Childhood, Myelodysplastic Syndrome(MDS)
Interventions
Romiplostim
Drug
Lead sponsor
Anjali Sharathkumar
Other
Eligibility
0 Years to 21 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jul 29, 2024 · Synced Jul 21, 2026, 11:48 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 Jul 21, 2026, 11:48 AM EDT