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Conditions: Down Syndrome, Chromosome Disorders, Intellectual and Developmental Disabilities
Interventions: Not listed
Lead sponsor: RTI International; Other
Eligibility: Not listed
Enrollment: 1,400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 15
States / cities: Orange, California • Aurora, Colorado • Miami, Florida + 12 more

Conditions: Atopic Dermatitis
Interventions: Dupilumab, Photo therapy; Drug · Other
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years to 60 Years
Enrollment: 7 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Scottsdale, Arizona

Conditions: Anemia, Sickle Cell
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 3,640 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 13
States / cities: Oakland, California • San Francisco, California • Boston, Massachusetts + 6 more

Conditions: Obsessive-Compulsive Disorder, Tic Disorders, Tourette Syndrome
Interventions: Ondansetron, Placebo; Drug
Lead sponsor: NYU Langone Health; Other
Eligibility: 18 Years to 60 Years
Enrollment: 110 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 2
States / cities: New York, New York

Conditions: Cystic Fibrosis
Interventions: micro Sweat Test Patch (mSTP), Sweat Testing using SOC; Device · Diagnostic Test
Lead sponsor: Emory University; Other
Eligibility: 2 Weeks to 90 Years
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 2
States / cities: Atlanta, Georgia

Conditions: Gout
Interventions: D-0120; Drug
Lead sponsor: InventisBio Co., Ltd; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 39 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2018
U.S. locations: 1
States / cities: Daytona Beach, Florida

Conditions: Down Syndrome, Dysphagia, Oropharyngeal
Interventions: Not listed
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 3 Years to 18 Years
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 1
States / cities: Aurora, Colorado

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: Type 1 Diabetes
Interventions: Measurement of vascular function per SphymgnoCor equipment; Procedure
Lead sponsor: Children's Mercy Hospital Kansas City; Other
Eligibility: 12 Years to 14 Years
Enrollment: 85 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2004 – 2007
U.S. locations: 1
States / cities: Kansas City, Missouri

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: Chronic Pain, Chiari Malformation
Interventions: Online Acceptance and Commitment Therapy Intervention; Behavioral
Lead sponsor: Kent State University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Kent, Ohio

Conditions: Respiratory Distress Syndrome, Newborn, Respiratory Distress Syndrome in Premature Infant
Interventions: Lung ultrasound; Diagnostic Test
Lead sponsor: Hackensack Meridian Health; Other
Eligibility: 27 Weeks to 34 Weeks
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Hackensack, New Jersey

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: End Stage Renal Disease, Arteriovenous Fistula, Arteriovenous Graft
Interventions: Arteriovenous Fistula (AVF), Arteriovenous Graft (AVG); Procedure
Lead sponsor: University of Pittsburgh; Other
Eligibility: 65 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

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

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Collaboration for Down Syndrome Progress (CDP)

IL-4 and Insulin Resistance for Treatment of Patients With Atopic Dermatitis

Establishing a Repository of Blood and DNA Samples From People With Sickle Cell Disease (Comprehensive Sickle Cell Centers Collaborative Genotype-Phenotype Database and Sample Repository)

Effects of Ondansetron in Obsessive-compulsive and Tic Disorders

Novel Point-of-care Sweat Chloride Testing Device for Monitoring CFTR Function

D-0120 Safety and PK/PD, Food Effect Study in Healthy Volunteers

Pulmonary Health in Children With Down Syndrome

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

Vascular Function in Adolescent, Diabetic Children

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

Online Acceptance and Commitment Therapy for Chronic Pain in Sample of People With Chiari Malformation

Utilization of Lung Ultrasound Score in Decision for Minimally Invasive Surfactant Administration

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

Trial of Fistula Versus Graft in Elderly Patients

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