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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

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: 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: 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

Conditions: Immune System Diseases
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: Not listed
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Severe Combined Immunodeficiency
Interventions: ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes; Drug
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: Not listed
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1990 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
Interventions: Venipuncture; Procedure
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Not listed
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2024
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Severe Combined Immunodeficiency
Interventions: AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan; Drug · Device
Lead sponsor: University of California, San Francisco; Other
Eligibility: 2 Months and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2038
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Severe Combined Immunodeficiency, X Linked, Gene Therapy
Interventions: autologous CD34+ cell transduced with G2SCID vector; Biological
Lead sponsor: David Williams; Other
Eligibility: 0 Years to 5 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2028
U.S. locations: 4
States / cities: Los Angeles, California • Atlanta, Georgia • Boston, Massachusetts + 1 more

Conditions: Severe Combined Immunodeficiency, T Cell Lymphocytopenia
Interventions: Not listed
Lead sponsor: University of California, San Francisco; Other
Eligibility: 1 Day to 30 Days
Enrollment: 1,800 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 2
States / cities: Chinle, Arizona • Tuba City, Arizona

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: Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions: Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning; Biological · Drug
Lead sponsor: Duke University; Other
Eligibility: Up to 21 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2014
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Severe Combined Immunodeficiency Disease, X-linked
Interventions: CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs; Genetic · Drug · Device
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 24 Months · Male only
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2034
U.S. locations: 3
States / cities: San Francisco, California • Memphis, Tennessee • Seattle, Washington

Conditions: Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper IgE Syndromes, Hyper IgM Deficiencies, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, Common Variable Immune Deficiency (CVID)
Interventions: CD3/CD19 negative allogeneic hematopoietic stem cells; Biological
Lead sponsor: Paul Szabolcs; Other
Eligibility: 5 Years to 45 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
Interventions: EZN-2279, Adagen; Biological
Lead sponsor: Leadiant Biosciences, Inc.; Industry
Eligibility: Not listed
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 6
States / cities: Los Angeles, California • San Francisco, California • Denver, Colorado + 3 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: 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: x Linked Combined Immunodeficiency
Interventions: CliniMACs; Device
Lead sponsor: University of Miami; Other
Eligibility: Up to 65 Years
U.S. locations: 1
States / cities: Miami, Florida

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: 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, 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

Conditions: Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions: BPX-501 and Rimiducid; Biological
Lead sponsor: Bellicum Pharmaceuticals; Industry
Eligibility: 4 Months to 55 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, ADA SCID, XSCID
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 690 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2028
U.S. locations: 39
States / cities: Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 34 more

Conditions: Ulcerative Colitis
Interventions: Tulisokibart, Companion Diagnostic (CDx) Testing, Placebo; Drug · Device · Other
Lead sponsor: Prometheus Biosciences, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA); Industry
Eligibility: 18 Years and older
Enrollment: 178 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 29
States / cities: Mobile, Alabama • Phoenix, Arizona • Sun City, Arizona + 24 more

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Human Placental-Derived Stem Cell Transplantation

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

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

Molecular and Clinical Studies of Primary Immunodeficiency Diseases

Developing Newborn Screening for Infants With Primary Immunodeficiency

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Autologous Gene Therapy for Artemis-Deficient SCID

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Newborn Screening for Severe Combined Immunodeficiency (SCID) in a High-Risk Population

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

EZN-2279 in Patients With ADA-SCID

Evaluation of Patients With Immune Function Abnormalities

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

cliniMACs HUD for T Cell Depletion

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

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

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Natural History Study of SCID Disorders

A Phase 2 Safety and Efficacy Study of Tulisokibart (MK-7240/PRA023) in Subjects With Moderately to Severely Active Ulcerative Colitis (MK-7240-005)