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Conditions: Dermatitis, Atopic
Interventions: Abrocitinib 100 mg, Abrocitinib 200 mg, Placebo; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years and older
Enrollment: 3,166 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 156
States / cities: Birmingham, Alabama • Anaheim, California • Encinitas, California + 101 more

Conditions: Cystinosis
Interventions: Cystagon®, RP103; Drug
Lead sponsor: Amgen; Industry
Eligibility: Not listed
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009
U.S. locations: 1
States / cities: San Diego, California

Conditions: Aicardi Syndrome, Brain Disorders
Interventions: Syndrome cause identification; Other
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Not listed
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2030
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: alglucosidase alfa, Placebo; Biological · Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 8 Years and older
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 5
States / cities: Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more

Conditions: Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration
Interventions: Gene Therapy Product-MCO-010, Sham Injection; Biological · Procedure
Lead sponsor: Nanoscope Therapeutics Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 5
States / cities: Beverly Hills, California • Pensacola, Florida • Fargo, North Dakota + 2 more

Conditions: Heart Defects, Congenital
Interventions: Clopidogrel (SR25990), placebo; Drug
Lead sponsor: Sanofi; Industry
Eligibility: Up to 92 Days
Enrollment: 906 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 1
States / cities: Bridgewater, New Jersey

Conditions: Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease, Charcot Marie Tooth Disease (CMT)
Interventions: Not listed
Lead sponsor: Hereditary Neuropathy Foundation; Government
Eligibility: Not listed
Enrollment: 10,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2029
U.S. locations: 1
States / cities: New York, New York

Conditions: X-Linked Myotubular Myopathy
Interventions: ASP2957, Methylprednisolone, Prednisolone, Sirolimus; Genetic · Drug
Lead sponsor: Astellas Gene Therapies; Industry
Eligibility: Up to 36 Months · Male only
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 4
States / cities: Chicago, Illinois • Boston, Massachusetts • Portland, Oregon + 1 more

Conditions: Retinitis Pigmentosa
Interventions: Implant of Argus II Retinal Prosthesis; Device
Lead sponsor: Second Sight Medical Products; Industry
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2019
U.S. locations: 7
States / cities: Los Angeles, California • San Francisco, California • Baltimore, Maryland + 3 more

Conditions: End Stage Renal Disease, Hyperhomocysteinemia
Interventions: cyanocobalamin, folic acid, pyridoxine; Drug
Lead sponsor: Georgetown University; Other
Eligibility: 21 Years to 89 Years
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2000
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Acute Undifferentiated Leukemia, 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), Mast Cell Leukemia, Myeloid/NK-cell Acute Leukemia, Untreated Adult Acute Lymphoblastic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions: bioelectric impedance analysis; Procedure
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: Not listed
Enrollment: 102 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 1
States / cities: Winston-Salem, North Carolina

Conditions: Neurofibromatosis 1
Interventions: Not listed
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years to 99 Years
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: AV Fistula
Interventions: Vorapaxar sulfate, Placebo; Drug
Lead sponsor: Ken Mahaffey; Other
Eligibility: 18 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Stanford, California

Conditions: Extensive Stage Small Cell Lung Cancer, Stage IIIA Non-small Cell Lung Cancer, Stage IV Non-small Cell Lung Cancer, Recurrent Small Cell Lung Cancer, Stage IIIB Non-small Cell Lung Cancer, Recurrent Non-small Cell Lung Cancer
Interventions: FR901228; Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Timeline: 2000 – 2007
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hypoplastic Left Heart Syndrome, Hypoplastic Right-sided Heart Complex
Interventions: Ambrisentan, Placebo; Drug · Other
Lead sponsor: Kevin Hill; Other
Eligibility: 24 Months to 120 Months
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Arterial Calcification, CD73 Deficiency
Interventions: Etidronate, MRI Scan; Drug · Device
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 80 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2021
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cystic Fibrosis, Methicillin-resistant Staphylococcus Aureus
Interventions: Vancomycin; Drug
Lead sponsor: Case Western Reserve University; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2021
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: NF2-related Schwannomatosis
Interventions: Auto-captioning Glasses; Device
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Vein of Galen Malformations
Interventions: Fetal embolization of vein of Galen malformation using Target XL and XXL Detachable Coils; Device
Lead sponsor: Darren Orbach; Other
Eligibility: 18 Years and older · Female only
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease
Interventions: Epeleuton; Drug
Lead sponsor: Afimmune; Industry
Eligibility: 18 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 16
States / cities: Birmingham, Alabama • Farmington, Connecticut • Washington D.C., District of Columbia + 11 more

Conditions: Niemann-Pick Disease, Type C
Interventions: IB1001; Drug
Lead sponsor: IntraBio Inc; Industry
Eligibility: 6 Years and older
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions: Group 1; Other
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 2 Years to 8 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2015
U.S. locations: 1
States / cities: New York, New York

Conditions: Sickle Cell Disease
Interventions: Alemtuzumab, low dose total body irradiation, Sirolimus; Drug
Lead sponsor: Robert Nickel; Other
Eligibility: 2 Years to 25 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2030
U.S. locations: 5
States / cities: Washington D.C., District of Columbia • Chicago, Illinois • New York, New York + 2 more

Conditions: Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome/Acute Myeloid Leukemia, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome, Recurrent Myelodysplastic Syndrome/Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Refractory Myelodysplastic Syndrome, Refractory Myelodysplastic Syndrome/Acute Myeloid Leukemia
Interventions: Biospecimen Collection, Bone Marrow Aspiration, Cirtuvivint, Decitabine and Cedazuridine, Echocardiography Test, Multigated Acquisition Scan; Procedure · Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 23
States / cities: New Haven, Connecticut • Atlanta, Georgia • Chicago, Illinois + 17 more

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Study to Evaluate Efficacy and Safety of PF-04965842 With or Without Topical Medications in Subjects Aged 12 Years and Older With Moderate to Severe Atopic Dermatitis

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Study of Selected X-Linked Disorders: Aicardi Syndrome

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

Efficacy And Safety Of Clopidogrel In Neonates /Infants With Systemic To Pulmonary Artery Shunt Palliation

Natural History Study for Charcot Marie Tooth Disease

Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators

Argus® II Retinal Stimulation System Feasibility Protocol

Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis

Bioelectrical Impedance Measurement for Predicting Treatment Outcome in Patients With Newly Diagnosed Acute Leukemia

Internet Support Group for Parents of a Child With Neurofibromatosis Type 1

Vorapaxar Study for Maturation of AV Fistulae for Hemodialysis Access

FR901228 in Treating Patients With Refractory or Progressive Small Cell Lung Cancer or Non-small Cell Lung Cancer

Ambrisentan in Single Ventricle

Etidronate for Arterial Calcifications Due to Deficiency in CD73 (ACDC)

Pharmacokinetics of Vancomycin for Inhalation in Cystic Fibrosis

Assessment of Patient Experience With Auto-Captioning Glasses in NF2-Related-Schwannomatosis

Fetal Treatment of Galenic Malformations

Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients With Sickle Cell Disease

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Collection of Cerebrospinal Fluid in Healthy Children

Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease

Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes