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ClinicalTrials.gov public records Last synced May 21, 2026, 6:19 PM EDT

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Showing 1–24 of 43 matching trials from the live ClinicalTrials.gov search.
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Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 21, 2026, 6:19 PM EDT
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 May 21, 2026, 6:19 PM EDT
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions
anti-thymocyte globulin, therapeutic allogeneic lymphocytes, fludarabine phosphate, thiotepa, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, in vitro-treated peripheral blood stem cell transplantation, total-body irradiation
Biological · Drug · Procedure + 1 more
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Year to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
2
States / cities
San Francisco, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 11, 2012 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions
Allogeneic stem cell transplant
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
Interventions
Treosulfan, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography
Drug · Biological · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 49 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
24
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 20 more
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
Drug · Other · Procedure + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
1 Year to 75 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Neonatal Thrombocytopenia
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
5 Days to 3 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2024
U.S. locations
3
States / cities
Iowa City, Iowa • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 15, 2024 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Cancer
Interventions
recombinant human thrombopoietin, carboplatin, etoposide, ifosfamide, G-CSF
Biological · Drug
Lead sponsor
Children's Oncology Group
Network
Eligibility
1 Year to 21 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2005
U.S. locations
23
States / cities
Long Beach, California • Los Angeles, California • Orange, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2014 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Grade III Lymphomatoid Granulomatosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Aplastic Anemia, Burkitt Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis, Childhood Immunoblastic Large Cell Lymphoma, Childhood Myelodysplastic Syndromes, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Juvenile Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Paroxysmal Nocturnal Hemoglobinuria, Peripheral T-cell Lymphoma, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Secondary Myelofibrosis, Severe Combined Immunodeficiency, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
fludarabine phosphate, melphalan, total-body irradiation, tacrolimus, mycophenolate mofetil, methotrexate, laboratory biomarker analysis, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Drug · Radiation · Other + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
3 Years to 75 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Sep 23, 2019 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Paroxysmal Nocturnal Hemoglobinuria, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia, Refractory Anemia With Ringed Sideroblasts, Relapsing Chronic Myelogenous Leukemia, Thrombocytopenia, Untreated Adult Acute Myeloid Leukemia
Interventions
bryostatin 1, sargramostim, laboratory biomarker analysis, pharmacological study
Drug · Biological · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 8, 2013 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP), Kawasaki Syndrome
Interventions
Immune Globulin Intravenous (Human), 10%
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
24 Months and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
11
States / cities
Los Angeles, California • Stanford, California • Englewood, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 23, 2021 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP)
Interventions
RAY121
Drug
Lead sponsor
Chugai Pharmaceutical
Industry
Eligibility
18 Years to 85 Years
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
11
States / cities
Orange, California • Baltimore, Maryland • Lake Success, New York + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
U.S. locations
14
States / cities
La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 6, 2018 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis, Blood Coagulation Disorder, Sickle Cell Disease, Dyskeratosis Congenita, Diamond-Blackfan Anemia, Congenital Thrombocytopenia, Severe Congenital Neutropenia, Fanconi Anemia, Myelodysplastic Syndromes, Myeloproliferative Diseases
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,716 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2050
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Multifocal Lymphangioendotheliomatosis With Thrombocytopenia, Cutaneovisceral Angiomatosis With Thrombocytopenia, Vascular Anomaly With Thrombocytopenia, Hemangiomas
Interventions
no intervention
Other
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2019
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 15, 2019 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Wiskott-Aldrich Syndrome (WAS), X-linked Thrombocytopenia
Interventions
Interleukin-2
Drug
Lead sponsor
Soma Jyonouchi
Other
Eligibility
24 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
3
States / cities
Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 15, 2017 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura, Familial Thrombotic Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Purpura, Congenital, Upshaw-Schulman Syndrome
Interventions
Observation
Other
Lead sponsor
Insel Gruppe AG, University Hospital Bern
Other
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2030
U.S. locations
1
States / cities
Oklahoma City, Oklahoma
Source: ClinicalTrials.gov public record
Updated Oct 10, 2023 · Synced May 21, 2026, 6:19 PM EDT
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 May 21, 2026, 6:19 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers
Conditions
Thrombocytopenia, Neonatal, Platelet Transfusion, Infant, Newborn, Diseases, Infant, Extremely Low Birth Weight, Infant, Small for Gestational Age, Thrombosis
Interventions
Higher Platelet Transfusion Threshold, Lower Platelet Transfusion Threshold
Procedure
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
1 Hour to 48 Hours
Enrollment
2,433 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2031
U.S. locations
20
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 17 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Fetal and Neonatal Alloimmune Thrombocytopenia
Interventions
Clinical data collection
Other
Lead sponsor
Rallybio
Industry
Eligibility
18 Years and older · Female only
Enrollment
14,390 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
19
States / cities
Chandler, Arizona • Gardena, California • New Haven, Connecticut + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Anemia, Thrombocytopenia, Coagulopathy, Mild, Hypofibrinogenemia
Interventions
Clinical Decision Support
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
1,543 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Mar 5, 2025 · Synced May 21, 2026, 6:19 PM EDT
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 May 21, 2026, 6:19 PM EDT
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced May 21, 2026, 6:19 PM EDT
Conditions
Neonatal Thrombocytopenia, Bleeding
Interventions
Not listed
Lead sponsor
Karolinska Institutet
Other
Eligibility
1 Day and older
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Boston, Massachusetts • Murray, Utah • Provo, Utah
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced May 21, 2026, 6:19 PM EDT