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Conditions: Sickle Cell Disease, Cerebral Stroke
Interventions: Carbon Dioxide; Other
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 6 Years to 70 Years
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2032
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Dermatitis, Dermatitis, Atopic, Eczema, Skin Diseases, Skin Diseases, Genetic, Genetic Diseases, Inborn, Skin Diseases, Eczematous, Hypersensitivity, Hypersensitivity, Immediate, Immune System Diseases
Interventions: PF-04965842 100 mg, PF-04965842 200 mg, Placebo; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years and older
Enrollment: 1,235 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 52
States / cities: Birmingham, Alabama • Fountain Valley, California • Fremont, California + 44 more

Conditions: Thyroid Eye Disease
Interventions: Teprotumumab; Biological
Lead sponsor: Amgen; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 8
States / cities: Beverly Hills, California • Los Angeles, California • Miami, Florida + 5 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 8
States / cities: Orlando, Florida • Iowa City, Iowa • East Lansing, Michigan + 5 more

Conditions: Sickle Cell Disease
Interventions: The adapted Kids in Transition to School Intervention, Standard school resources; Behavioral
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 42 Months to 78 Months
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Pompe Disease
Interventions: ATB200, AT2221; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2024
U.S. locations: 12
States / cities: Phoenix, Arizona • Orange, California • Gainesville, Florida + 9 more

Conditions: Microsatellite Stable (MSS) Colorectal Adenocarcinomas, Colorectal Adenocarcinoma
Interventions: Nivolumab, Relatlimab; Drug
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: 18 Years and older
Enrollment: 59 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Sickle Cell Disease
Interventions: SCD Group; Other
Lead sponsor: University Hospitals Cleveland Medical Center; Other
Eligibility: 12 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2030
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Cystic Fibrosis
Interventions: Initiation of CFTR Modulator, Hyperpolarized Xenon 129; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 6 Years to 21 Years
Enrollment: 26 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2026
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Hematologic Diseases, Anaemia, Sickle Cell
Interventions: GSK4172239D, Placebo; Drug · Other
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 6
States / cities: Miami, Florida • Tamarac, Florida • Atlanta, Georgia + 3 more

Conditions: Cystic Fibrosis
Interventions: N91115; Drug
Lead sponsor: Nivalis Therapeutics, Inc.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 19
States / cities: Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 15 more

Conditions: Bloom Syndrome
Interventions: Not listed
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 2 Years to 55 Years
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: New York, New York

Conditions: Eczema Atopic Dermatitis, Eczema, Atopic Dermatitis
Interventions: GX-03, Vehicle; Combination Product · Other
Lead sponsor: Turn Therapeutics; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 190 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Irving, Texas

Conditions: Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
Interventions: Not listed
Lead sponsor: Splice Bio; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 14
States / cities: San Diego, California • Aurora, Colorado • Gainesville, Florida + 10 more

Conditions: Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 6
States / cities: Palo Alto, California • Aurora, Colorado • Boston, Massachusetts + 3 more

Conditions: Attenuated Familial Adenomatous Polyposis, Colorectal Carcinoma, Duodenal Carcinoma, Familial Adenomatous Polyposis
Interventions: Biopsy Procedure, Biospecimen Collection, Gastrointestinal Endoscopy, Obeticholic Acid, Placebo Administration, Questionnaire Administration; Procedure · Biological · Drug + 1 more
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 6
States / cities: Scottsdale, Arizona • Kansas City, Kansas • Boston, Massachusetts + 3 more

Conditions: Huntington Disease
Interventions: Not listed
Lead sponsor: Huntington Study Group; Network
Eligibility: 18 Years and older
Enrollment: 600 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Rochester, New York

Conditions: Retinitis Pigmentosa
Interventions: Intravitreal autologous CD34+ cells; Biological
Lead sponsor: University of California, Davis; Other
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Sacramento, California

Conditions: Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions: Clofarabine, Fludarabine Injection; Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 17 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Fragile X Syndrome
Interventions: BPN14770/ zatolmilast, Placebo; Drug
Lead sponsor: Tetra Discovery Partners; Industry
Eligibility: 18 Years to 45 Years · Male only
Enrollment: 171 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 17
States / cities: Irvine, California • Orange, California • Sacramento, California + 14 more

Conditions: Duchenne Muscular Dystrophy
Interventions: RGX-202; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 1 Year and older · Male only
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2028
U.S. locations: 19
States / cities: Little Rock, Arkansas • Orange, California • Palo Alto, California + 16 more

Conditions: Sickle Cell Disease
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 80 Years
Enrollment: 99 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cystic Fibrosis
Interventions: Sildenafil 40mg oral capsule, Placebo Oral capsule; Drug
Lead sponsor: National Jewish Health; Other
Eligibility: 9 Years and older
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 2
States / cities: Denver, Colorado • Augusta, Georgia

Conditions: Neurofibromatosis Type 1, Reading Disabilities
Interventions: Tutoring Program I, Tutoring Program II; Behavioral
Lead sponsor: Vanderbilt University; Other
Eligibility: 8 Years to 17 Years
Enrollment: 184 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2017
U.S. locations: 1
States / cities: Nashville, Tennessee

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Cerebrovascular Reactivity Measurements With High-Density Diffuse Optical Tomography

Study to Investigate Efficacy and Safety of PF-04965842 in Subjects Aged 12 Years and Over With Moderate to Severe Atopic Dermatitis With the Option of Rescue Treatment in Flaring Subjects

Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Study

Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A

School Readiness Intervention for Preschool Children With Sickle Cell Disease

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Study of Nivolumab and Relatlimab in Patients With Microsatellite Stable (MSS) Advanced Colorectal Cancer

Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)

Non-contrast Lung Perfusion Mapping Applied for New Insights in Cystic Fibrosis

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years

Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation

Biological Significance of the Bloom's Syndrome Protein

A Double-Blind, Randomized, Vehicle-Controlled Phase 2 Study to Assess the Efficacy and Safety of GX-03 in Adult Subjects With Moderate to Severe Atopic Dermatitis (Eczema)

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

Testing Obeticholic Acid for Familial Adenomatous Polyposis

Longitudinal Endpoint Assessment of Disease Burden in HD

Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Pathophysiology of Acute Pain in Patients With Sickle Cell Disease

Sildenafil Exercise: Role of PDE5 Inhibition

Neurobiology and Treatment of Reading Disability in NF-1