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Conditions: Substance Use Disorders, Pregnancy Related, Alcohol Use Disorder (AUD), Alcohol Use Complicating Pregnancy, First Trimester, Alcohol Use Complicating Pregnancy, Second Trimester, Alcohol Use Complicating Pregnancy, Third Trimester, Alcohol Use Complicating Pregnancy, Unspecified Trimester, Alcohol Use Complicating Pregnancy, Childbirth, and the Puerperium, Fetal Alcohol Spectrum Disorders, Fetal Alcohol Syndrome, Drug Use Disorders, Drug Use Complicating Pregnancy, First Trimester, Drug Use Complicating Pregnancy, Second Trimester, Drug Use Complicating Pregnancy, Third Trimester, Drug Use Complicating Pregnancy, Unspecified Trimester, Drug Use Complicating Pregnancy, Childbirth, and the Puerperium, Maternal Drugs Affecting Fetus
Interventions: Parent-Child Assistance Program; Other
Lead sponsor: University of Oklahoma; Other
Eligibility: 18 Years and older · Female only
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 2
States / cities: Oklahoma City, Oklahoma • Tulsa, Oklahoma

Conditions: Atopic Dermatitis
Interventions: Upadacitinib, Dupilumab; Drug
Lead sponsor: AbbVie; Industry
Eligibility: 2 Years to 11 Years
Enrollment: 699 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 31
States / cities: Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 27 more

Conditions: Prader Willi Syndrome, Syndromic Obesity, Childhood Obesity
Interventions: Low Carbohydrate diet, Low Fat diet; Other
Lead sponsor: Duke University; Other
Eligibility: 5 Years to 17 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Osteoarthritis, Rheumatoid Arthritis, Femoral Fracture, Correction of a Functional Deformity, Avascular Necrosis
Interventions: Encore Linear™ Hip System; Device
Lead sponsor: Encore Medical, L.P.; Industry
Eligibility: 18 Years and older
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2011
U.S. locations: 1
States / cities: Vero Beach, Florida

Conditions: Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy 4, SMA
Interventions: Spinal Cord Stimulator (Octopolar Medtronic Vectris Leads); Device
Lead sponsor: Marco Capogrosso; Other
Eligibility: 16 Years to 65 Years
Enrollment: 3 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Cystinuria
Interventions: Bucillamine; Drug
Lead sponsor: Revive Therapeutics, Ltd.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 5
States / cities: Birmingham, Alabama • Boston, Massachusetts • New York, New York + 2 more

Conditions: Degenerative Disc Disease, Herniated Disc, Spinal Stenosis, Spondylolithesis, Spinal Deformity, Cervical Myelopathy, Failed Back Surgery Syndrome, Spinal Cord Neoplasms
Interventions: Bone graft substitute, autograft or allograft; Device
Lead sponsor: Baxter Healthcare Corporation; Industry
Eligibility: 18 Years and older
Enrollment: 329 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 12
States / cities: San Francisco, California • Atlanta, Georgia • Carrollton, Georgia + 9 more

Conditions: Lowe Syndrome
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: Male only
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2009
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
Interventions: Open Label Topical Beremagene Geperpavec (B-VEC); Biological
Lead sponsor: Krystal Biotech, Inc.; Industry
Eligibility: 2 Months and older
Enrollment: 47 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 6
States / cities: Rancho Santa Margarita, California • Redwood City, California • Coral Gables, Florida + 3 more

Conditions: Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD, High Cholesterol
Interventions: Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe, Combination Therapy placebo, Obicetrapib Placebo, Ezetimibe Placebo; Combination Product · Drug · Other
Lead sponsor: NewAmsterdam Pharma; Industry
Eligibility: 18 Years and older
Enrollment: 407 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024
U.S. locations: 45
States / cities: Gilbert, Arizona • Jacksonville, Florida • Port Orange, Florida + 37 more

Conditions: Tourette Syndrome
Interventions: Not listed
Lead sponsor: University of Utah; Other
Eligibility: 18 Years to 35 Years · Male only
Enrollment: 21 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2012
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Interventions: INZ-701; Drug
Lead sponsor: Inozyme Pharma; Industry
Eligibility: Up to 1 Year
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 3
States / cities: San Diego, California • Columbus, Ohio • Philadelphia, Pennsylvania

Conditions: Hypothalamic Obesity, Multiple Pituitary Hormone Deficiency Genetic Form, Septo-Optic Dysplasia, Optic Nerve Hypoplasia, Childhood-onset Combined Pituitary Hormone Deficiency, Pituitary Stalk Interruption Syndrome
Interventions: Setmelanotide, Placebo; Drug
Lead sponsor: Rhythm Pharmaceuticals, Inc.; Industry
Eligibility: 4 Years and older
Enrollment: 39 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 8
States / cities: Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 5 more

Conditions: Alport Syndrome, Focal Segmental Glomerulosclerosis
Interventions: R3R01; Drug
Lead sponsor: River 3 Renal Corp.; Industry
Eligibility: 12 Years and older
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 14
States / cities: Los Angeles, California • Boca Raton, Florida • Miami, Florida + 11 more

Conditions: Gout
Interventions: Storytelling; Behavioral
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 18 Years and older
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 4
States / cities: Birmingham, Alabama • Iowa City, Iowa • Boston, Massachusetts + 1 more

Conditions: Cystic Fibrosis
Interventions: Inhaled Vancomycin, Placebo (Sterile Water), Rifampin, Trimethoprim/Sulfamethoxazole (TMP/SMX), Doxycycline, Mupirocin Intranasal Creme, 4% chlorhexidine gluconate liquid skin cleanser; Drug
Lead sponsor: Johns Hopkins University; Other
Eligibility: 12 Years and older
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2017
U.S. locations: 2
States / cities: Baltimore, Maryland • Cleveland, Ohio

Conditions: Juvenile Idiopathic Arthritis
Interventions: Tocilizumab; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2019
U.S. locations: 2
States / cities: Los Angeles, California • Cincinnati, Ohio

Conditions: Tuberous Sclerosis Complex, Lymphangioleiomyomatosis
Interventions: Not listed
Lead sponsor: David M. Ritter; Other
Eligibility: Female only
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions: Not listed
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Akron, Ohio

Conditions: Cardiac Death, Cardiac Death, Sudden, Wolf Parkinson White Syndrome
Interventions: Ablation, Standard of care without ablation; Procedure
Lead sponsor: United States Naval Medical Center, San Diego; Federal
Eligibility: 18 Years to 40 Years
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2025
U.S. locations: 1
States / cities: San Diego, California

Conditions: Fabry Disease
Interventions: Migalastat HCl 20 mg; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 2 Years to 11 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 6
States / cities: Atlanta, Georgia • Minneapolis, Minnesota • Charlotte, North Carolina + 3 more

Conditions: Iron Overload, Thalassemia
Interventions: Combination Iron Chelation Therapy; Drug
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: 7 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2002
U.S. locations: 1
States / cities: New York, New York

Conditions: Aortopathies, Thoracic Aortic Aneurysm, Aortic Valve Disease, Thoracic Aortic Disease, Thoracic Aortic Dissection, Thoracic Aortic Rupture, Ascending Aortic Disease, Descending Aortic Disease, Ascending Aortic Aneurysm, Descending Aortic Aneurysm, Marfan Syndrome, Loeys-Dietz Syndrome, Vascular Ehlers-Danlos Syndrome, Shprintzen-Goldberg Syndrome, Turner Syndrome, PHACE Syndrome, Autosomal Recessive Cutis Laxa, Congenital Contractural Arachnodactyly, Arterial Tortuosity Syndrome, Bicuspid Aortic Valve-Associated Aortopathy, Bicuspid Aortic Valve, Familial Thoracic Aortic Aneurysm and Aortic Dissection
Interventions: Not listed
Lead sponsor: Yale University; Other
Eligibility: Not listed
Enrollment: 3,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2030
U.S. locations: 2
States / cities: Atlanta, Georgia • Indianapolis, Indiana

Conditions: Huntington Disease
Interventions: Fenofibrate, Placebo; Drug
Lead sponsor: University of California, Irvine; Other
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 1
States / cities: Irvine, California

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The Oklahoma Parent-Child Assistance Program

A Study to Assess Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Children From 2 to Less Than 12 Years of Age With Moderate to Severe Atopic Dermatitis

Prader-Willi Syndrome Macronutrient Study

Retrospective Study of the Linear™ Hip

Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

Bucillamine Phase 2 Trial in Patients With Cystinuria

Bone Graft Materials Observational Registry

Genetic Analysis of Oculocerebrorenal Syndrome of Lowe

A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa

Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies

MRI Study of Tic Remission in Tourette Syndrome

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis

Gout in the ED and Improving Research Participation

Persistent Methicillin Resistant Staphylococcus Aureus Eradication Protocol (PMEP)

A Study of Decreased Dose Frequency in Participants With Systemic Juvenile Arthritis Who Experience Laboratory Abnormalities During Treatment With RoActemra/Actemra (Tocilizumab)

Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Longitudinal Evaluation of Active-Duty Personnel with Accessory Pathways (LEAD-AP)

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Combination Iron Chelation Therapy

Pathogenetic Basis of Aortopathy and Aortic Valve Disease

Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease