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Conditions: Cystic Fibrosis
Interventions: Not listed
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 12 Years and older
Enrollment: 54 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2024
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania

Conditions: Myotonic Dystrophy Type 1
Interventions: Not listed
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years and older
Enrollment: 88 participants
Timeline: 2022 – 2028
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Retinitis Pigmentosa
Interventions: Not listed
Lead sponsor: National Eye Institute (NEI); NIH
Eligibility: Not listed
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1993 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

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: Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions: HDAC inhibitor AR-42, decitabine, laboratory biomarker analysis, pharmacological study; Drug · Other
Lead sponsor: Alison Walker; Other
Eligibility: 3 Years and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 3
States / cities: Cincinnati, Ohio • Columbus, Ohio

Conditions: Anakinra, Kineret, Atopic Dermatitis, Eczema, Allergic Disease
Interventions: Anakinra; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 10 Years to 30 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Parkinson's Disease, Parkinsonism, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy, Shy-Drager Syndrome, Corticobasal Degeneration, Dementia With Lewy Bodies, Pick Disease, Olivopontocerebellar Atrophies
Interventions: Not listed
Lead sponsor: Bastyr University; Other
Eligibility: 19 Years and older
Enrollment: 2,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2030
U.S. locations: 1
States / cities: Kenmore, Washington

Conditions: Hemophilia A
Interventions: BIIB031 (rFVIIIFc), FVIII (PK subgroup only); Drug
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: Up to 11 Years · Male only
Enrollment: 71 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 12
States / cities: Los Angeles, California • Sacramento, California • San Diego, California + 9 more

Conditions: Epilepsy, Tuberous Sclerosis Complex
Interventions: Everolimus; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 2 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2016
U.S. locations: 2
States / cities: Cincinnati, Ohio • Houston, Texas

Conditions: Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Interventions: Bone Marrow Aspiration and Biopsy, Trametinib; Procedure · Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 1 Month to 21 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 57
States / cities: Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more

Conditions: XMEN
Interventions: Magnesium L-threonate, Placebo, Intravenous (IV) magnesium sulfate (MgSO4); Dietary Supplement · Other · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 6 Years and older · Male only
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cystic Fibrosis
Interventions: Xylitol, Hypertonic saline; Drug
Lead sponsor: Joseph Zabner; Other
Eligibility: 12 Years and older
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 1
States / cities: Iowa City, Iowa

Conditions: Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions: Thiotepa; Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 17 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Congenital Heart Disease
Interventions: Device-Aided performances, Device-Unaided performances; Device · Other
Lead sponsor: BrightHeart; Industry
Eligibility: 18 Years and older · Female only
Enrollment: 200 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024
U.S. locations: 1
States / cities: New York, New York

Conditions: Tourette Syndrome
Interventions: Valbenazine; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 6 Years to 64 Years
Enrollment: 155 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 13
States / cities: Sun City, Arizona • San Diego, California • Loxahatchee Groves, Florida + 10 more

Conditions: Generalized Lipodystrophy
Interventions: Mibavademab, Placebo; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 2
States / cities: Ann Arbor, Michigan • Dallas, Texas

Conditions: Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
Interventions: AR-42; Drug
Lead sponsor: Massachusetts Eye and Ear Infirmary; Other
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2021
U.S. locations: 4
States / cities: Stanford, California • Baltimore, Maryland • Boston, Massachusetts + 1 more

Conditions: Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Interventions: Ultrase® MT20, Placebo; Drug
Lead sponsor: Forest Laboratories; Industry
Eligibility: 7 Years and older
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2007
U.S. locations: 4
States / cities: Grand Rapids, Michigan • Cleveland, Ohio • Hershey, Pennsylvania + 1 more

Conditions: Atopic Dermatitis
Interventions: GHZ339, Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years to 100 Years
Enrollment: 224 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 24
States / cities: Birmingham, Alabama • Fort Smith, Arkansas • Fountain Valley, California + 20 more

Conditions: TOURETTE SYNDROME
Interventions: SD-809; Drug
Lead sponsor: Auspex Pharmaceuticals, Inc.; Industry
Eligibility: 12 Years to 18 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
Interventions: Burosumab; Drug
Lead sponsor: Laura Tosi; Other
Eligibility: Not listed
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Umbilical Hernia
Interventions: Infra-umbilical incision, Transumbilical incision; Procedure
Lead sponsor: Children's Mercy Hospital Kansas City; Other
Eligibility: Up to 9 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025
U.S. locations: 1
States / cities: Kansas City, Missouri

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: Male only
Enrollment: 69 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 6
States / cities: Mobile, Alabama • Torrance, California • Peoria, Illinois + 3 more

Conditions: Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
Interventions: AR882 Dose 1, AR882 Dose 2, Allopurinol Tablet; Drug
Lead sponsor: Arthrosi Therapeutics; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 10
States / cities: Birmingham, Alabama • Phoenix, Arizona • Sun City, Arizona + 7 more

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Muscle Accrual and Function in Cystic Fibrosis-Impact of Glucose Intolerance

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

Study of Usher Syndromes, Type 1 and Type 2

Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts

AR-42 and Decitabine in Treating Patients With Acute Myeloid Leukemia

A Pilot Study Using Anakinra/Kineret for the Treatment of Patients With Severe Atopic Dermatitis

Modifiable Variables in Parkinsonism (MVP)

Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A

Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

Magnesium Supplementation in People With XMEN Syndrome

Aerosolized Hypertonic Xylitol Versus Hypertonic Saline in Cystic Fibrosis (CF) Subjects

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

Fetal Heart Ultrasound Suspicious Radiographic Finding Identification - Reader Study

Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome

Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy

Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma

Safety and Efficacy Study of ULTRASE® MT20 in Participants With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)

Platform Study to Evaluate the Efficacy and Safety of Investigational Compound(s) in Patients With Moderate to Severe Atopic Dermatitis

A Pilot Study Of SD-809 (Deutetrabenazine) In Moderate To Severe Tourette Syndrome (TS)

Burosumab for CSHS

Cosmetic Outcomes of Umbilical Hernia Incisions

Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A

Phase 2 to Assess Efficacy and Safety in AR882 Alone or in Combination With Allopurinol in Patients With Tophaceous Gout