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Conditions: Atopic Dermatitis
Interventions: Dupilumab, Midazolam, Omeprazole, Warfarin, Caffeine, Metoprolol; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 5
States / cities: Little Rock, Arkansas • Centennial, Colorado • Minneapolis, Minnesota + 2 more

Conditions: MPS-IH (Hurler Syndrome)
Interventions: Experimental: OTL-203, Active Comparator: Allo-HSCT; Genetic
Lead sponsor: Orchard Therapeutics; Industry
Eligibility: 28 Days to 30 Months
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2031
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome, Complete Atypical DiGeorge Syndrome
Interventions: Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw, Rabbit anti-thymocyte globulin, Cyclosporine, Tacrolimus, Methylprednisolone or Prednisolone, Daclizumab, Mycophenolate mofetil; Biological · Other · Procedure + 1 more
Lead sponsor: Sumitomo Pharma Switzerland GmbH; Industry
Eligibility: Not listed
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2017
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Atopic Dermatitis (AD), Asthma
Interventions: dupilumab; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 18 Years and older · Female only
Enrollment: 581 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2025
U.S. locations: 1
States / cities: La Jolla, California

Conditions: Alpha1-Antitrypsin Deficiency
Interventions: No Intervention; Other
Lead sponsor: Takeda; Industry
Eligibility: 18 Years and older
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2031
U.S. locations: 3
States / cities: Gainesville, Florida • Charleston, South Carolina • Nashville, Tennessee

Conditions: Hemophilia A
Interventions: Antihemophilic factor, recombinant, manufactured protein-free; Drug
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 5 Years and older · Male only
Enrollment: 59 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2004
U.S. locations: 10
States / cities: Los Angeles, California • Atlanta, Georgia • Indianapolis, Indiana + 7 more

Conditions: Smith Lemli Opitz Syndrome, CHILD Syndrome, Lathosterolosis, Desmosterolosis
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 1 Day to 100 Years
Enrollment: 250 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2031
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Giant Cell Arteritis, Takayasu's Arteritis, Polyarteritis Nodosa, Wegener's Granulomatosis, Microscopic Polyangiitis, Churg-Strauss Syndrome, Behcet's Disease, Kawasaki Disease, Henoch-schoenlein Purpura, Vasculitis, Central Nervous System, Drug-induced Necrotizing Vasculitis
Interventions: Not listed
Lead sponsor: University of South Florida; Other
Eligibility: 18 Years and older
Enrollment: 467 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis, Becker Muscular Dystrophy, Heart Failure With Preserved Ejection Fraction, Heart Failure With Reduced Ejection Fraction
Interventions: Hyperpolarized 13C-Pyruvate; Diagnostic Test
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 18 Years to 60 Years
Enrollment: 7 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Cystic Fibrosis
Interventions: Vitamin D Powder, Vitamin D Oil; Dietary Supplement
Lead sponsor: Emory University; Other
Eligibility: 18 Years to 59 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Autosomal Dominant Polycystic Kidney Disease
Interventions: Tolvaptan MR, Tolvaptan IR, Placebo; Drug
Lead sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 178 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2013
U.S. locations: 35
States / cities: Huntsville, Alabama • Mobile, Alabama • Peoria, Arizona + 31 more

Conditions: Cytomegalovirus Infection
Interventions: Valganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 99 Years
Enrollment: 205 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 6
States / cities: Los Angeles, California • Atlanta, Georgia • Rochester, Minnesota + 3 more

Conditions: Primary Immune Deficiency
Interventions: Human Normal Immunoglobulin for Subcutaneous Administration; Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 2 Years to 75 Years
Enrollment: 49 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 13
States / cities: Los Angeles, California • Centennial, Colorado • North Palm Beach, Florida + 9 more

Conditions: Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions: Bone marrow transplantation, Mesenchymal Stromal Cells; Procedure · Biological
Lead sponsor: Stanford University; Other
Eligibility: 1 Year to 25 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 2
States / cities: Birmingham, Alabama • Minneapolis, Minnesota

Conditions: Gaucher Disease
Interventions: Not listed
Lead sponsor: O & O Alpan LLC; Other
Eligibility: 18 Years and older
Enrollment: 33 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2016
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Alpha 1 Antitrypsin Deficiency
Interventions: Alpha-1 15%, Liquid Alpha1-PI; Drug · Other
Lead sponsor: Grifols Therapeutics LLC; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 10
States / cities: Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 7 more

Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions: Not listed
Lead sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.; Industry
Eligibility: 12 Years to 70 Years
Enrollment: 3,409 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 102
States / cities: Peoria, Arizona • Tempe, Arizona • Azusa, California + 89 more

Conditions: Sickle Cell Disease
Interventions: Alemtuzumab, Hematopoietic Cell Transplantation, Intensity-Modulated Radiation Therapy, Sirolimus; Drug · Procedure · Radiation
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 12 Years to 40 Years
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Duarte, California

Conditions: Healthy Volunteers
Interventions: Teprotumumab; Drug
Lead sponsor: Amgen; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 44 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023
U.S. locations: 1
States / cities: Las Vegas, Nevada

Conditions: HIV Infections, Hemophilia A
Interventions: Zidovudine; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 12 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1989
U.S. locations: 2
States / cities: Buffalo, New York • Rochester, New York

Conditions: Usher Syndrome, Retinitis Pigmentosa
Interventions: Not listed
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 0 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 1999
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Fuchs' Endothelial Dystrophy
Interventions: Netarsudil, Placebo; Drug
Lead sponsor: Price Vision Group; Industry
Eligibility: 18 Years and older
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Indianapolis, Indiana

Conditions: Advanced Unresectable Melanoma, Metastatic Melanoma
Interventions: Mirdametinib; Drug
Lead sponsor: Kevin Kim, MD; Other
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Spinocerebellar Ataxia Type 3
Interventions: varenicline, placebo; Drug
Lead sponsor: University of South Florida; Other
Eligibility: 18 Years to 80 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 3
States / cities: Los Angeles, California • Gainesville, Florida • Tampa, Florida

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Open Label, Drug-Drug Interaction (DDI) Study of Dupilumab (REGN668/SAR231893) in Patients With Moderate to Severe Atopic Dermatitis (AD)

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Phase I/II Thymus Transplantation With Immunosuppression #950

Post-authorization Safety Study in North America to Monitor Pregnancy and Infant Outcomes Following Administration of Dupilumab During Planned or Unexpected Pregnancy

A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems

Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery

Biochemical and Phenotypical Aspects of Smith-Lemli-Opitz Syndrome and Related Disorders of Cholesterol Metabolism

Reproductive Health in Men and Women With Vasculitis

UTSW HP [13-C] Pyruvate Injection in HCM

Comparison of Absorption of Vitamin D in Cystic Fibrosis

8-Week Study of Tolvaptan Dose Forms in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

CMV Antiviral Prevention Strategies in D+R-Liver Transplants ("CAPSIL")

Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy

Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

Exploration of Immunity in Gaucher Disease

A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)

Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease

TMLI and Alemtuzumab for Treatment of Sickle Cell Disease

A Trial to Investigate Teprotumumab (High-concentration Formulation) Subcutaneous Administration in Healthy Adult Non-Japanese and Japanese Participants

Safety and Effectiveness of Azidothymidine (AZT) in HIV-Positive Patients With Hemophilia

Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher Syndrome

Trial of Netarsudil for Acceleration of Corneal Endothelial Restoration

Mirdametinib in Patients With Advanced NF1-mutant Melanoma

Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3