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Conditions: Hematologic Malignancies
Interventions: UCB and HPDSC; Procedure
Lead sponsor: Celgene Corporation; Industry
Eligibility: Up to 55 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2013
U.S. locations: 1
States / cities: New Orleans, Louisiana

Conditions: Lymphoma, Non-Hodgkin, HIV Infections
Interventions: Filgrastim, Vincristine sulfate, Doxorubicin hydrochloride, Cyclophosphamide, Cytarabine, Dexamethasone; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 16 Years and older
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1998
U.S. locations: 7
States / cities: San Francisco, California • Denver, Colorado • Boston, Massachusetts + 3 more

Conditions: Adenosine Deaminase Deficiency, ADA-SCID
Interventions: Lentiviral Gene Transfer; Genetic
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Year to 65 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Bethesda, Maryland

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: 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: 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: 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: Severe Combined Immunodeficiency
Interventions: Anti-thymocyte globulin (rabbit), Busulfan, Fludarabine, Thiotepa, CliniMACS, Donor Lymphocyte Infusion; Drug · Device · Other
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 2 Months and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: SCID, ADA-SCID, XSCID, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 1,007 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2023
U.S. locations: 31
States / cities: Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 26 more

Conditions: Severe Combined Immunodeficiency
Interventions: Stem cell transplant, Filgrastim, Alemtuzumab, Miltenyi CliniMACS; Procedure · Drug · Device
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 2 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2007
U.S. locations: 1
States / cities: Memphis, Tennessee

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: Pneumonia, Pneumocystis Carinii, HIV Infections
Interventions: Atovaquone; Drug
Lead sponsor: Glaxo Wellcome; Industry
Eligibility: 1 Day and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: RTI International; Other
Eligibility: 1 Day to 31 Days
Enrollment: 30,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

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: Sarcoma, Kaposi, HIV Infections
Interventions: Doxorubicin hydrochloride (liposomal), Bleomycin sulfate, Vincristine sulfate, Doxorubicin hydrochloride; Drug
Lead sponsor: Sequus Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 225 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 28
States / cities: Berkeley, California • Beverly Hills, California • Encino, California + 16 more

Conditions: Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
Interventions: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV); Biological
Lead sponsor: University of California, Los Angeles; Other
Eligibility: Not listed
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2040
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: Primary Immunodeficiency, DOCK8, Virus Susceptibility
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years to 85 Years
Enrollment: 200 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2009
U.S. locations: 1
States / cities: Bethesda, Maryland

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: 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: 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 Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2), Coronavirus Disease 2019 (COVID-19), Immunocompromised, Hospitalization, Child, Hospitalized
Interventions: Nirmatrelvir, Ritonavir, Placebo for nirmatrelvir; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 2
States / cities: Cooperstown, New York • New York, New York

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: 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

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A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

Phase II Trial of Sequential Chemotherapy and Radiotherapy for AIDS-Related Primary Central Nervous System Lymphoma

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

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

Autologous Gene Therapy for Artemis-Deficient SCID

Human Placental-Derived Stem Cell Transplantation

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

Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)

Patients Treated for SCID (1968-Present)

Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

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

An Open-Label, Uncontrolled Clinical Trial of Oral 566C80 for the Treatment of Patients With Severe PCP Who Are Intolerant and/or Unresponsive to Therapy With Trimethoprim/Sulfamethoxazole and Parenteral Pentamidine

Early Check: Expanded Screening in Newborns

The Role of Bacteria and Genetic Variations in Cystic Fibrosis

A Comparison of DOX-SL Versus Adriamycin Plus Bleomycin Plus Vincristine in the Treatment of Severe AIDS-Related Kaposi's Sarcoma

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

cliniMACs HUD for T Cell Depletion

Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders

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

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

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

A Study to Learn About the Medicine Called Nirmatrelvir Used in Combination With Ritonavir in People With Weakened Immune Systems or at Increased Risk for Poor Outcomes Who Are Hospitalized Due to Severe COVID-19

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

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