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Conditions: Dermatomyositis, Glycogen Storage Disease Type II, Glycogen Storage Disease Type VII, Myositis, Polymyositis
Interventions: Not listed
Lead sponsor: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); NIH
Eligibility: Not listed
Healthy volunteers: Accepts healthy volunteers
Timeline: 1993 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 4
States / cities: Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Up to 24 Months
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2021
U.S. locations: 3
States / cities: Gainesville, Florida • Decatur, Georgia • Detroit, Michigan

Conditions: Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII, Tarui Disease, GSD X, GSD XII, GSD XIII, GSD XV, PGBM2, PRKAG2, Danon Disease, Polyglucosan Body Myopathy Type 1, Polyglucosan Body Myopathy Type 2, RBCK1 Deficiency
Interventions: No intervention; Other
Lead sponsor: Duke University; Other
Eligibility: 0 Years to 90 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2034
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Glycogen Storage Disease Type II
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 8 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 3
States / cities: Washington D.C., District of Columbia • St Louis, Missouri • Seattle, Washington

Conditions: Glycogen Storage Disease Type Ia
Interventions: No Intervention; Other
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 2 Years and older
Enrollment: 140 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2036
U.S. locations: 9
States / cities: Orange, California • Denver, Colorado • Hartford, Connecticut + 6 more

Conditions: Age-related Macular Degeneration
Interventions: Ranibizumab 0.5mg; Drug
Lead sponsor: Southern California Desert Retina Consultants, MC; Other
Eligibility: 50 Years and older
Enrollment: 108 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 4
States / cities: Mountain View, California • Palm Desert, California • Baltimore, Maryland + 1 more

Conditions: Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions: AB-1009 (GAA Gene); Genetic
Lead sponsor: AskBio Inc; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 9
States / cities: Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more

Conditions: Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Up to 12 Months
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 14
States / cities: Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more

Conditions: Glycogen Storage Disease VI, GLYCOGEN STORAGE DISEASE IXa1, GLYCOGEN STORAGE DISEASE IXa2, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, GSD 9 (All Subtypes), GSD 6
Interventions: No intervention; Other
Lead sponsor: Duke University; Other
Eligibility: 0 Years to 90 Years
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2030
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 16 Years and older · Female only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 1
States / cities: San Antonio, Texas

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: Age-Related Macular Degeneration, Macular Degeneration
Interventions: Intravitreal Aflibercept Injection; Drug
Lead sponsor: MidAtlantic Retina; Other
Eligibility: 18 Years and older
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 3
States / cities: Cherry Hill, New Jersey • Huntingdon Valley, Pennsylvania • Philadelphia, Pennsylvania

Conditions: Pompe Disease
Interventions: Albuterol; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Epilepsy, GLUT1DS1, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome
Interventions: Triheptanoin; Drug
Lead sponsor: Juan Pascual; Other
Eligibility: 2 Years to 35 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
Interventions: GZ402666; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 7
States / cities: Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 4 more

Conditions: Pompe Disease
Interventions: rAAV1-CMV-GAA (study agent) Administration, RMST; Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 2 Years to 18 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions: Whole-body Electrical Muscle Stimulation Exercise; Device
Lead sponsor: University of Missouri-Columbia; Other
Eligibility: 18 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 1
States / cities: Columbia, Missouri

Conditions: Pompe Disease
Interventions: Thoracic MRI, Spirometry, Inspiratory Load Compensation, Maximal Inspiratory Pressure, Resting Breathing Pattern, Respiratory Muscle Endurance Test; Other
Lead sponsor: University of Florida; Other
Eligibility: 20 Years to 65 Years
Enrollment: 14 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Lysosomal Storage Disorders
Interventions: Not listed
Lead sponsor: O & O Alpan LLC; Other
Eligibility: 1 Day to 100 Years
Enrollment: 80 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2022
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Pompe Disease (Late-onset)
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease, Glycogen Storage Disease Type II, Lysosomal Storage Diseases
Interventions: Diet and Exercise; Other
Lead sponsor: University of Florida; Other
Eligibility: 15 Years to 55 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Pompe Disease
Interventions: S-606001, Placebo; Drug
Lead sponsor: Shionogi; Industry
Eligibility: 18 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 9
States / cities: Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more

Conditions: Pompe Disease
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: 0 Years to 18 Years
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2029
U.S. locations: 1
States / cities: Durham, North Carolina

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Genetic and Family Studies of Inherited Muscle Diseases

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Growth and Development Study of Alglucosidase Alfa

Rare Glycogen Storage Diseases Natural History Study

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Prophylactic Ranibizumab for Exudative Age-related Macular Degeneration

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

GSD VI and GSD IX Natural History

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

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

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

Treatment Development of Triheptanoin (G1D)

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Pompe Gene Therapy- Screening for Eligibility

Diet and Exercise in Pompe Disease

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Cognitive and Neurological Pathologies in Pompe Disease