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Showing 1–17 of 17 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Autoimmune Pancytopenia Clear all
Completed Phase 1Phase 2 Interventional Results available

Sirolimus for Autoimmune Disease of Blood Cells

NCT00392951
View directory record Official record
Conditions
Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS), Evans Syndrome, Idiopathic Thrombocytopenic Purpura, Anemia, Hemolytic, Autoimmune, Autoimmune Neutropenia, Lupus Erythematosus, Systemic, Inflammatory Bowel Disease, Rheumatoid Arthritis
Interventions
sirolimus
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Year to 30 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 18, 2019 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 2 Interventional Results available

Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia

NCT00260689
View directory record Official record
Conditions
Immunosuppresion, Thrombocytopenia, Pancytopenia, Neutropenia
Interventions
Anti-thymocyte globulin (rabbit), Anti-thymocyte globulin (horse), Cyclosporine, Alemtuzumab
Biological · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 7, 2017 · Synced Jul 22, 2026, 3:45 AM 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 Jul 22, 2026, 3:45 AM EDT
Recruiting No phase listed Observational

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

NCT01351545
View directory record Official record
Conditions
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia
Interventions
A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Drug
Lead sponsor
Center for International Blood and Marrow Transplant Research
Network
Eligibility
Not listed
Enrollment
99,999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2041
U.S. locations
142
States / cities
Birmingham, Alabama • Gilbert, Arizona • Phoenix, Arizona + 90 more
Source: ClinicalTrials.gov public record
Updated Jul 5, 2026 · Synced Jul 22, 2026, 3:45 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 Jul 22, 2026, 3:45 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jul 22, 2026, 3:45 AM 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 22, 2026, 3:45 AM EDT
Completed Phase 2 Interventional

Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia

NCT00005935
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Mycophenolate mofetil, Cyclosporine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 22, 2026, 3:45 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 22, 2026, 3:45 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 Jul 22, 2026, 3:45 AM EDT
Completed Phase 2 Interventional Results available

Eltrombopag for Moderate Aplastic Anemia

NCT01328587
View directory record Official record
Conditions
Moderate Aplastic Anemia, Unilineage Bone Marrow Failure Disorders
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

NCT01623167
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Cohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6, Extension Cohort
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 95 Years
Enrollment
207 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting Phase 2 Interventional Results available

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

NCT04304820
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 99 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Not yet recruiting Phase 2 Interventional

Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases

NCT07660783
View directory record Official record
Conditions
Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia, Immune Dysregulation, Hemophagocytic Lymphohistiocytosis (HLH), Non Malignant Disorders
Interventions
ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Biological
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
6 Months to 50 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
2
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 21, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 2 Interventional Results available

Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia

NCT00065260
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Campath-1H, r-ATG, CsA
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 20, 2021 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Not applicable Interventional

Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders

NCT01758042
View directory record Official record
Conditions
Chronic Kidney Disease, Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Chronic Myelogenous Leukemia (CML), Chronic Lymphocytic Leukemia (CLL), Non-Hodgkin's Lymphoma (NHL), Hodgkin Disease, Multiple Myeloma, Myelodysplastic Syndrome (MDS), Aplastic Anemia, AL Amyloidosis, Diamond Blackfan Anemia, Myelofibrosis, Myeloproliferative Disease, Sickle Cell Anemia, Autoimmune Diseases, Thalassemia
Interventions
Haploidentical Bone Marrow/Kidney
Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Jul 22, 2026, 3:45 AM EDT