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ClinicalTrials.gov public records Last synced May 21, 2026, 8:08 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 33 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, 8:08 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, 8:08 PM EDT
Conditions
Immunologic Deficiency Syndromes
Interventions
Immune Globulin Intravenous (Human) Omr-IgG-am IGIV
Drug
Lead sponsor
FFF Enterprises
Industry
Eligibility
3 Years to 75 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
8
States / cities
Los Angeles, California • Centennial, Colorado • North Palm Beach, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2014 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 2 Interventional
Conditions
Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
Interventions
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced May 21, 2026, 8:08 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, 8:08 PM EDT
Conditions
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Immunoproliferative Disorder
Interventions
Bacteriophage, rhuCD40L, KLH
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Melanoma (Skin)
Interventions
MART-1 antigen, aldesleukin, gp100 antigen, recombinant CD40-ligand, recombinant interferon gamma, recombinant interleukin-4, sargramostim, therapeutic autologous dendritic cells, therapeutic tumor infiltrating lymphocytes, tyrosinase peptide, Candida albicans skin test reagent
Biological · Radiation
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2006
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 21, 2014 · Synced May 21, 2026, 8:08 PM EDT
Conditions
X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome, Wiskott-Aldrich Syndrome, Leukocyte Adhesion Deficiency Syndrome
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Esophageal Neoplasms
Interventions
CD40 Gene Transfer
Genetic
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
2
States / cities
Ridgewood, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated May 2, 2016 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
Up to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 14, 2009 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Leukemia
Interventions
Dose Level 1, Dose Level 2, Dose Level 3
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 75 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
3
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 20, 2020 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Type 1 Diabetes Mellitus
Interventions
Islet transplantation with Tegoprubart (AT-1501) immunosupression-based therapy
Drug
Lead sponsor
University of Chicago
Other
Eligibility
18 Years to 65 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Wiskott- Aldrich Syndrome, ADA Deficient SCID
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
3 Years and older
Enrollment
266 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Chronic Lymphocytic B-Leukemia
Interventions
Injection of IL-2-secreting CD40L-expressing autologous B-CLL cells
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2010
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 22, 2012 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinaemia, Hyperimmunoglobulin M Syndrome
Interventions
Subgam
Biological
Lead sponsor
Bio Products Laboratory
Other
Eligibility
2 Years to 75 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
16
States / cities
Chandler, Arizona • Irvine, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2018 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Leukemia, Leukemia, B-Cell, Chronic
Interventions
Dose Level 1, Dose Level 2, Dose Level 2- Fixed Dose
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2010
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 20, 2020 · Synced May 21, 2026, 8:08 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers
Conditions
Acute Bacterial Infections, Acute Viral Infections
Interventions
Not listed
Lead sponsor
The Plasma Source
Industry
Eligibility
18 Years to 70 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Southampton, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 19, 2012 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome, Wiskott-Aldrich Syndrome
Interventions
Gammaplex
Biological
Lead sponsor
Bio Products Laboratory
Other
Eligibility
2 Years to 16 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
8
States / cities
Irvine, California • Los Angeles, California • Centennial, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2014 · Synced May 21, 2026, 8:08 PM EDT
Conditions
SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
Interventions
Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan, Stem Cell Transplantation, Fludarabine phosphate 40 mg, Melphalan, Alemtuzumab 0.2 mg, Fludarabine phosphate 30 mg, MESNA
Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced May 21, 2026, 8:08 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, 8:08 PM EDT
Conditions
Lupus Erythematosus, Systemic
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
13 Years and older
Healthy volunteers
Accepts healthy volunteers
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Melanoma, Non-small Cell Lung Cancer, Breast Cancer, Gastric Cancer, Renal Cell Carcinoma, Ovarian Cancer, Cholangiocarcinoma, Bladder Urothelial Carcinoma, Pancreatic Adenocarcinoma, Colorectal Cancer, Esophageal Cancer, Hepatic Cancer, Head and Neck Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer, Other Solid Tumors, Diffuse Large B-cell Lymphoma (DLBCL), Mantle Cell Lymphoma, Indolent B-cell Lymphomas, Non-Hodgkin Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Waldenstrom's Disease, Marginal Zone Lymphoma, Mucosa Associated Lymphoid Tissue, Small Lymphocytic Leukemia
Interventions
CDX-1140, CDX-301, pembrolizumab, Chemotherapy
Drug
Lead sponsor
Celldex Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
11
States / cities
Scottsdale, Arizona • Atlanta, Georgia • Augusta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2024 · Synced May 21, 2026, 8:08 PM EDT
Conditions
Hyper-IgM Syndrome, Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
119 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2017 · Synced May 21, 2026, 8:08 PM EDT