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Conditions: Granulomatous Disease, Chronic
Interventions: Busulfan; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2010
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hereditary Angioedema Types I and II
Interventions: Ecallantide subcutaneous dosing; Drug
Lead sponsor: NYU Langone Health; Other
Eligibility: 2 Years to 16 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Mineola, New York

Conditions: Primary Immune Deficiency
Interventions: Subcutaneous IgG (SCIG); Biological
Lead sponsor: CSL Behring; Industry
Eligibility: Up to 2 Years
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010
U.S. locations: 5
States / cities: Los Angeles, California • Tampa, Florida • Plymouth, Minnesota + 2 more

Conditions: Colitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 21 Years and older · Male only
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Primary Immune Deficiency
Interventions: Newnorm; Biological
Lead sponsor: Octapharma; Industry
Eligibility: 2 Years to 75 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 10
States / cities: Irvine, California • Centennial, Colorado • Port Saint Lucie, Florida + 7 more

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

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: Ebola Virus Disease, Marburg Virus Disease
Interventions: cAd3-Marburg, cAd3-EBO-S; Biological
Lead sponsor: Albert B. Sabin Vaccine Institute; Other
Eligibility: 18 Years to 50 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021
U.S. locations: 1
States / cities: Oklahoma City, Oklahoma

Conditions: Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, HAE
Interventions: ADX-324 Dose Level 1, ADX-324 Dose Level 2; Drug
Lead sponsor: ADARx Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 15
States / cities: Litchfield Park, Arizona • Little Rock, Arkansas • San Diego, California + 12 more

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Roche Global Development; Other
Eligibility: 13 Years and older
Enrollment: 450 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 23
States / cities: Beverly Hills, California • Irvine, California • Los Angeles, California + 16 more

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

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Foscarnet sodium; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 13 Years to 65 Years
Enrollment: 156 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1992
U.S. locations: 11
States / cities: Los Angeles, California • San Francisco, California • Miami, Florida + 7 more

Conditions: Primary Immune Deficiency Disorders (PIDD)
Interventions: Nabi-IGIV 10% [Immune Globulin Intravenous (Human). 10%]; Biological
Lead sponsor: ADMA Biologics, Inc.; Industry
Eligibility: 6 Years to 75 Years
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 17
States / cities: Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 13 more

Conditions: Microtia, Dysbiosis, Alcohol Drinking, HIV Infections, Cardiovascular Diseases
Interventions: Probiotic, Placebo; Dietary Supplement
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: 18 Years to 89 Years
Enrollment: 250 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Foscarnet sodium; Drug
Lead sponsor: Astra USA; Industry
Eligibility: 18 Years to 65 Years
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Hereditary Angioedema
Interventions: NTLA-2002, Normal Saline IV Administration; Biological
Lead sponsor: Intellia Therapeutics; Industry
Eligibility: 16 Years and older
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 14
States / cities: Scottsdale, Arizona • San Diego, California • Santa Monica, California + 11 more

Conditions: Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
Interventions: Stem Cell Transplant, Myeloablative conditioning regimen; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 55 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2015
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Colitis, HIV Infections
Interventions: Glutamic acid hydrochloride, Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 13 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1998
U.S. locations: 5
States / cities: Birmingham, Alabama • San Francisco, California • St Louis, Missouri + 2 more

Conditions: Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
Interventions: Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Sirolimus, Tacrolimus, Total-Body Irradiation; Procedure · Drug · Other + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Up to 55 Years
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2019
U.S. locations: 3
States / cities: Nashville, Tennessee • Seattle, Washington

Conditions: Obesity, Immune Deficiency
Interventions: Institutional standard intravenous immune globulin treatment; Drug
Lead sponsor: Rutgers, The State University of New Jersey; Other
Eligibility: 18 Years to 75 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Somerville, New Jersey

Conditions: Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT), Primary Immunodeficiency Disorders (PID)
Interventions: Norovirus -specific T-cell (NST) therapy; Biological
Lead sponsor: Children's National Research Institute; Other
Eligibility: 3 Months to 80 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2028
U.S. locations: 3
States / cities: Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland

Conditions: Primary Immunodeficiency Diseases (PID)
Interventions: SC treatment with IGSC, 10% with rHuPH20 followed by SC/IGSC, 10% only (safety), SC treatment with IGSC, 10% with rHuPH20 followed by IV/IGSC, 10% only (safety), IV treatment with IGSC, 10%; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 2 Years and older
Enrollment: 66 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 11
States / cities: Cypress, California • Irvine, California • Los Angeles, California + 8 more

Conditions: Down Syndrome
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 1 Year to 120 Years
Enrollment: 700 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2040
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Zidovudine, Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 8
States / cities: Birmingham, Alabama • Oakland, California • San Francisco, California + 5 more

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Use of Busulfan as Conditioning Agent for a Second Stem Cell Transplant

Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema

Retrospective Chart Review of Subcutaneous IgG Use in Infants

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

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

Evaluation of Safety, Tolerability and Immune Responses of Ebola-S and Marburg Vaccines in Healthy Adults

Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)

A Randomized, Controlled Study of the Safety and Preventive Efficacy of Oral Ganciclovir When Used in Conjunction With An Intravitreal Ganciclovir Implant in the Treatment of Cytomegalovirus Retinitis

Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM Syndrome

A Phase II Dose-Ranging, Open-Labelled Trial of Foscarnet Salvage Therapy for AIDS Patients With Sight-Threatening CMV Retinitis Who Cannot Be Treated With Ganciclovir Due To Myelosuppression or Treatment Failure

Open Label, Phase III Study of NABI-IGIV 10% [Immune Globulin Intravenous(Human), 10%] In Subjects With Primary Immune Deficiency Disorders (PIDD)

Microbiome Metabolites and Alcohol in HIV to Reduce CVD RCT

A Study of Foscarnet in the Treatment of Cytomegalovirus (CMV) of the Eyes in Patients With AIDS Who Have Not Had Success With Ganciclovir

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

A Randomized Comparative Pharmacokinetic Study of Oral Ganciclovir After Treatment With Intravenous Ganciclovir for Cytomegalovirus Gastrointestinal Disease in AIDS Patients

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

Immunoglobulin G Therapy Dose Optimization

Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts

Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PID

Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy

A Study of the Safety and Tolerance of Long-Term Therapy With Intravenous Cytovene (Ganciclovir Sodium) for Cytomegalovirus Retinitis in Persons With AIDS