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Conditions: Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
Interventions: Fludarabine, Cyclophosphamide, Busulfan, Alemtuzumab, Rabbit Anti-thymocyte Globulin, Horse Anti-thymocyte Globulin; Drug
Lead sponsor: Columbia University; Other
Eligibility: Up to 30 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2021
U.S. locations: 1
States / cities: New York, New York

Conditions: Adenosine Deaminase Deficiency, Autosomal Recessive Disorder, Immune System Disorder, Purine-Nucleoside Phosphorylase Deficiency, Severe Combined Immunodeficiency, Severe Combined Immunodeficiency With Absence of T and B Cells, X-Linked Severe Combined Immunodeficiency
Interventions: Allogeneic Bone Marrow Transplantation, Cyclosporine, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation; Procedure · Drug · Other + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Not listed
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2018
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Psuedomas Infection, Cystic Fibrosis
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 9 Years to 80 Years
Enrollment: 76 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2018
U.S. locations: 4
States / cities: Bethesda, Maryland • Seattle, Washington • Madison, Wisconsin + 1 more

Conditions: Epilepsy
Interventions: USL261, Placebo; Drug
Lead sponsor: UCB Biopharma S.P.R.L.; Industry
Eligibility: 12 Years and older
Enrollment: 292 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2017
U.S. locations: 46
States / cities: Phoenix, Arizona • Tucson, Arizona • Little Rock, Arkansas + 43 more

Conditions: SCID
Interventions: Busulfan, Cell processing for TCRαβ+/CD19+ depletion; Drug · Device
Lead sponsor: Center for International Blood and Marrow Transplant Research; Network
Eligibility: 0 Years to 2 Years
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2028
U.S. locations: 37
States / cities: Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 32 more

Conditions: Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 3,500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2005
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions: Hematopoietic stem cell transplant (HSCT); Biological
Lead sponsor: Paul Szabolcs; Other
Eligibility: 5 Years to 40 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

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

Conditions: Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions: OTL-101; Biological
Lead sponsor: University of California, Los Angeles; Other
Eligibility: 30 Days to 17 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
Interventions: Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells; Genetic · Drug · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 3 Years to 99 Years · Male only
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2034
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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

Conditions: Severe Combined Immunodeficiency Syndrome
Interventions: CD34+ cells transduced with ADA retrovir; Drug
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2014
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT; Genetic · Drug
Lead sponsor: University of California, Los Angeles; Other
Eligibility: 30 Days to 17 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Severe Combined Immunodeficiency
Interventions: Transplant Conditioning with Mobilization Only, Transplant Conditioning with Mobilization and Alemtuzumab; Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 3 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 1
States / cities: San Francisco, California

Conditions: SCID
Interventions: Humanized anti-CD117 Monoclonal Antibody (JSP191); Biological
Lead sponsor: Jasper Therapeutics, Inc.; Industry
Eligibility: 3 Months to 12 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2025
U.S. locations: 9
States / cities: Los Angeles, California • Palo Alto, California • San Francisco, California + 6 more

Conditions: Alopecia
Interventions: FK506, GP11046, GP11511; Drug
Lead sponsor: Hordinsky, Maria K., MD; Individual
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 35 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2003 – 2004
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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

Conditions: Severe Combined Immunodeficiency
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 13 Years to 17 Years · Female only
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: T-Cell Immune Deficiency Diseases, Severe Combined Immunodeficiency
Interventions: Busulfan, Fludarabine and ATG; Drug
Lead sponsor: Emory University; Other
Eligibility: Not listed
Healthy volunteers: Accepts healthy volunteers
Timeline: Ends 2006
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Immunologic Deficiency Syndrome, Agammaglobulinemia, Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome, Common Variable Immunodeficiency
Interventions: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water; Drug
Lead sponsor: Grifols Therapeutics LLC; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2004
U.S. locations: 9
States / cities: Birmingham, Alabama • Denver, Colorado • Washington D.C., District of Columbia + 6 more

Conditions: Leukemia, Aplastic Anemia, Bone Marrow Failure Syndromes, SCID
Interventions: PCA plus methadone (Experimental), PCA Alone (SOC); Drug
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 6 Years to 18 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2028 – 2029
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions: CliniMACS CD34 Reagent System; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: Up to 65 Years
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Mesothelioma, Malignant
Interventions: Placebo, Amatuximab, Pemetrexed, Cisplatin; Drug
Lead sponsor: Morphotek; Industry
Eligibility: 18 Years and older
Enrollment: 124 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 7
States / cities: La Jolla, California • Newark, Delaware • Bethesda, Maryland + 4 more

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Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases

Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant

The Role of Bacteria and Genetic Variations in Cystic Fibrosis

Study to Evaluate the Safety and Efficacy of USL261 (Intranasal Midazolam) in Patients With Seizure Clusters

Conditioning SCID Infants Diagnosed Early

Evaluation of Patients With Immune Function Abnormalities

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

Immune Disorder HSCT Protocol

A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

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

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim

JSP191 Antibody Targeting Conditioning in SCID Patients

Effects of Ligands on Human Hair Follicles Grafted Onto SCID Mice

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID

IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency

Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients

Methadone as an Alternative Treatment for Children Underdoing HSCT

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Study of the Safety and Efficacy of Amatuximab in Combination With Pemetrexed and Cisplatin in Subjects With Unresectable Malignant Pleural Mesothelioma (MPM)