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Showing 1–24 of 320 matching trials from the live ClinicalTrials.gov search.
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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 Jul 23, 2026, 12:33 PM EDT
Terminated Phase 2 Interventional Results available

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

NCT03600909
View directory record Official record
Conditions
Fanconi Anemia, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Interventions
Busulfan, Fludarabine, Cyclophosphamide, Anti-Thymocyte Globulin (Rabbit), The CliniMACS device, G-CSF
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Month and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
New York, New York • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Jul 23, 2026, 12:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Extended Platelet Parameters as a Means to Differentiate Immune Thrombocytopenia From Hypo-proliferative Thrombocytopenias.

NCT01933035
View directory record Official record
Conditions
Immune Thrombocytopenia, Chemotherapy Induced Thrombocytopenia, Myelodysplasia, Aplastic Anaemia
Interventions
Immune Thrombocytopenics, Hypo-proliferative thrombocytopenics, Control Population
Other
Lead sponsor
Beth Israel Medical Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 16, 2016 · Synced Jul 23, 2026, 12:33 PM EDT
Recruiting Phase 2 Interventional

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

NCT04356469
View directory record Official record
Conditions
Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
Interventions
Haploidentical Hematopoietic Cell Transplantation
Biological
Lead sponsor
Johns Hopkins All Children's Hospital
Other
Eligibility
0 Years to 21 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
St. Petersburg, Florida
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Jul 23, 2026, 12:33 PM EDT
Recruiting Early Phase 1 Interventional

Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies

NCT06398457
View directory record Official record
Conditions
Hematologic Malignancy, Bone Marrow Transplant Rejection, Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS), Acute Lymphoblastic Leukemia (ALL), Adult, Multiple Myeloma, Aplastic Anemia, Lymphoma, Non Hodgkin Lymphoma, Hodgkin Lymphoma, Chronic Myeloid Leukemia, Myelofibrosis
Interventions
Darzalex Faspro (Daratumumab and hyaluronidase-fihj), JH-DSA Semi-Quant Screen and Response Score
Drug · Device
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 25, 2025 · Synced Jul 23, 2026, 12:33 PM EDT
Completed Phase 2 Interventional Results available

A Study of LY2606368 (Prexasertib) in Patients With Solid Tumors With Replicative Stress or Homologous Repair Deficiency

NCT02873975
View directory record Official record
Conditions
Advanced Cancers
Interventions
LY2606368
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 20, 2022 · Synced Jul 23, 2026, 12:33 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Treatment of Menorrhagia in Women With Thrombocytopenia Using Platelets or Platelets and Hormones

NCT00556400
View directory record Official record
Conditions
Aplastic Anemia, Menorrhagia, Amenorrhea
Interventions
Lo-Ovral Oral Contraceptive Pills, Placebo - sugar pill
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
12 Years to 55 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 18, 2013 · Synced Jul 23, 2026, 12:33 PM EDT
Recruiting No phase listed Observational

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

NCT07649031
View directory record Official record
Conditions
Fanconi Anemia
Interventions
MRIs
Diagnostic Test
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Jul 23, 2026, 12:33 PM EDT
Terminated Not applicable Interventional Results available

G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome

NCT00228813
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
Granulocyte Colony Stimulating Factor
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 22 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2015
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 10, 2017 · Synced Jul 23, 2026, 12:33 PM EDT
Active, not recruiting Phase 2 Interventional

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

NCT02979873
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Sirolimus
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Jul 23, 2026, 12:33 PM EDT
Completed Phase 1 Interventional

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

NCT00578266
View directory record Official record
Conditions
Anemia, Aplastic
Interventions
Cyclophosphamide,Campath IH and TBI
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
Up to 60 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 23, 2017 · Synced Jul 23, 2026, 12:33 PM 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 Jul 23, 2026, 12:33 PM 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 Jul 23, 2026, 12:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

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

NCT00964821
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions
trivalent influenza vaccine, Immunoenzyme technique, Laboratory biomarker analysis
Biological · Other
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 120 Years
Enrollment
459 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2013
U.S. locations
2
States / cities
Duarte, California • Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 6, 2017 · Synced Jul 23, 2026, 12:33 PM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

NCT01383434
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Bone Marrow Transplant
Procedure
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
6 Months to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 12, 2016 · Synced Jul 23, 2026, 12:33 PM EDT
Completed Phase 1 Interventional

Haplo Peripheral Blood Sct In GVHD Prevention

NCT04473911
View directory record Official record
Conditions
GVHD, AML, ALL, MDS, MPN, CMML, Hodgkin Lymphoma, Non Hodgkin Lymphoma, Blood Stem Cell Transplant Failure, Graft Vs Host Disease, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Myeloproliferative Disorder, Chronic Myelomonocytic Leukemia, Chemosensitive Hodgkin Lymphoma
Interventions
FLUDARABINE, CYCLOPHOSPHAMIDE, TBI, Melphalan, Sirolimus, Mycophenolate mofetil, RGI-2001
Drug · Radiation
Lead sponsor
Zachariah Michael DeFilipp
Other
Eligibility
18 Years to 80 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 19, 2025 · Synced Jul 23, 2026, 12:33 PM EDT
Terminated Not applicable Interventional

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

NCT00290628
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, filgrastim, melphalan, methylprednisolone, mycophenolate mofetil, radiation therapy, umbilical cord blood transplantation
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 45 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2007
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 28, 2017 · Synced Jul 23, 2026, 12:33 PM EDT
Completed Phase 2 Interventional Results available

Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States

NCT00997386
View directory record Official record
Conditions
Hematologic Neoplasms, Multiple Myeloma, Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Myelofibrosis
Interventions
busulfan, and melphalan, and alemtuzumab
Drug
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 75 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 8, 2019 · Synced Jul 23, 2026, 12:33 PM 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 Jul 23, 2026, 12:33 PM EDT
Recruiting No phase listed Observational

Familial Investigations of Childhood Cancer Predisposition

NCT03050268
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 23, 2026, 12:33 PM EDT
Not yet recruiting No phase listed Observational

Clinical Genetics Branch Eligibility Screening Survey

NCT07005297
View directory record Official record
Conditions
Melanoma, Li-Fraumeni Syndrome, Pulmonary Blastoma, Chordoma, Congenital Bone Marrow Failure Syndromes, Costello Syndrome, Fanconi Anemia, CFC Syndrome (CFCS), Legius Syndrome, RASopathies
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 99 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Jul 23, 2026, 12:33 PM 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 Jul 23, 2026, 12:33 PM EDT
Completed No phase listed Observational

A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia

NCT07415083
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Jul 23, 2026, 12:33 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Alefacept in Patients With Relapsed/Refractory Aplastic Anemia

NCT01267643
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Alefacept
Drug
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 25, 2018 · Synced Jul 23, 2026, 12:33 PM EDT