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Showing 1–24 of 319 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Bone Marrow Failure Syndromes Clear all
Completed Phase 3 Interventional Results available

Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder

NCT00755040
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
cyclosporine ophthalmic emulsion, placebo
Drug · Other
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
18 Years and older
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
5
States / cities
Palo Alto, California • Chicago, Illinois • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2017 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Phase 1Phase 2 Interventional

Conditioning Regimens for Patients With Severe Aplastic Anemia Transplanted With Marrow From an Unrelated Donor

NCT00144729
View directory record Official record
Conditions
Anemia, Aplastic
Interventions
Bone marrow transplant
Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2007
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 29, 2008 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Phase 1Phase 2 Interventional

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

NCT00777231
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
3
States / cities
Louisville, Kentucky • Durham, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 13, 2020 · Synced Sep 9, 2026, 1:54 AM EDT
Recruiting Not applicable Interventional

Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy

NCT06689800
View directory record Official record
Conditions
Hematologic Malignancy, Solid Tumor, Sickle Cell Disease, Aplastic Anemia, Immune Deficiency, Metabolic Disorder
Interventions
Let's Get REAL
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
8 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Phase 2 Interventional Results available

Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

NCT03476330
View directory record Official record
Conditions
Fanconi Anemia, Squamous Cell Carcinoma
Interventions
Quercetin (dietary supplement)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 19, 2026 · Synced Sep 9, 2026, 1:54 AM EDT
Not yet recruiting Phase 1 Interventional

Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

NCT07585136
View directory record Official record
Conditions
Bone Marrow Failure Syndrome
Interventions
Filgrastim, Plerixafor, Leukapheresis
Drug · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
18 Years to 25 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 30, 2026 · Synced Sep 9, 2026, 1:54 AM EDT
Completed No phase listed Observational

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609827
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Melphalan
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 3, 2021 · Synced Sep 9, 2026, 1:54 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 Jul 3, 2025 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Phase 2 Interventional Results available

NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

NCT02833805
View directory record Official record
Conditions
Severe Aplastic Anemia, Aplastic Anemia, Bone Marrow Failure, Immunosuppression
Interventions
Thymoglobulin, Fludarabine, Cyclophosphamide, Total body irradiation, Tacrolimus, Mycophenolate mofetil
Drug · Radiation
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 11, 2022 · Synced Sep 9, 2026, 1:54 AM EDT
Terminated Phase 1 Interventional

Cyclophosphamide, Antithymocyte Globulin, and Total-Body Irradiation in Treating Patients With Severe Aplastic Anemia Undergoing Umbilical Cord Blood Transplant

NCT00354419
View directory record Official record
Conditions
Aplastic Anemia
Interventions
total-body irradiation, cyclophosphamide, anti-thymocyte globulin, cyclosporine, umbilical cord blood transplantation, mycophenolate mofetil, bone marrow aspiration, DNA analysis, filgrastim
Radiation · Drug · Biological + 2 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 40 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 4, 2011 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Phase 2 Interventional

Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

NCT00004464
View directory record Official record
Conditions
Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Interventions
cyclophosphamide, filgrastim
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Up to 70 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 17, 2019 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Not applicable Interventional Results available

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

NCT02162420
View directory record Official record
Conditions
Dyskeratosis Congenita, Aplastic Anemia
Interventions
Alemtuzumab, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem Cell Transplant, Anti-thymocyte globulin
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 70 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 7, 2025 · Synced Sep 9, 2026, 1:54 AM 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 9, 2026, 1:54 AM EDT
Completed Phase 2 Interventional

Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors

NCT00618969
View directory record Official record
Conditions
Hematologic Neoplasms, Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Multiple Myeloma
Interventions
haploidentical allogeneic PBSC transp
Other
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 75 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Mar 2, 2016 · Synced Sep 9, 2026, 1:54 AM 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 9, 2026, 1:54 AM EDT
Completed Not applicable Interventional

Peripheral Blood Stem Cell Transplantation Using the CliniMACS Device

NCT01071226
View directory record Official record
Conditions
Malignant Diseases (ie, Leukemia, MDS, Lymphoma), Non-malignant Diseases (ie, Bone Marrow Failure Syndromes)
Interventions
CliniMACS (CD+3, CD+19 depletion), CliniMACS (CD 34+ positive selection), HSCT
Device · Other
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Up to 22 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Oct 24, 2021 · Synced Sep 9, 2026, 1:54 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 Sep 9, 2026, 1:54 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

NCT03192397
View directory record Official record
Conditions
Acute Myeloid Leukemia in Remission, Adult Acute Lymphoblastic Leukemia in Complete Remission, Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission, Chronic Myelomonocytic Leukemia in Remission, Graft Versus Host Disease, Hodgkin Lymphoma, Minimal Residual Disease, Myelodysplastic Syndrome, Myeloproliferative Neoplasm, Non-Hodgkin Lymphoma, Plasma Cell Myeloma, Severe Aplastic Anemia, Waldenstrom Macroglobulinemia
Interventions
Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Melphalan Hydrochloride, Mycophenolate Mofetil, Sirolimus, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

NCT00987480
View directory record Official record
Conditions
Aplastic Anemia, Leukemia, Myelodysplastic Syndrome
Interventions
Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine., CliniMACS device
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
6
States / cities
Boston, Massachusetts • New York, New York • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2018 · Synced Sep 9, 2026, 1:54 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Diabetes/ Endocrine Surveillance in SDS

NCT04275479
View directory record Official record
Conditions
Shwachman-Diamond Syndrome
Interventions
Oral Glucose Tolerance Test, Modified Oral Glucose Tolerance Test, Modified Mixed Meal Tolerance Test, Continuous Glucose Monitor, Food Diary, Medical History Questionnaires
Diagnostic Test · Other · Device
Lead sponsor
Washington University School of Medicine
Other
Eligibility
3 Years and older
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 18, 2024 · Synced Sep 9, 2026, 1:54 AM EDT
Completed Phase 2 Interventional Results available

Alemtuzumab to Treat Severe Aplastic Anemia

NCT00195624
View directory record Official record
Conditions
Severe Aplastic Anemia, Refractory, Severe Aplastic Anemia, Relapse
Interventions
Alemtuzumab (Campath ), Cyclosporine
Biological · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 110 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 6, 2020 · Synced Sep 9, 2026, 1:54 AM EDT
Completed No phase listed Observational

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609840
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Thiotepa
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 20, 2023 · Synced Sep 9, 2026, 1:54 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 Aug 5, 2026 · Synced Sep 9, 2026, 1:54 AM EDT
Recruiting No phase listed Observational

Shwachman Diamond Syndrome Registry and Study

NCT06056908
View directory record Official record
Conditions
Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
5,000 participants
Timeline
2016 – 2090
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Sep 9, 2026, 1:54 AM EDT