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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: Pompe Disease
Interventions: BMN 701; Biological
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 13 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 4
States / cities: San Diego, California • Gainesville, Florida • Tampa, Florida + 1 more

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: Pompe Disease
Interventions: Clenbuterol, Placebo; Drug
Lead sponsor: Dwight Koeberl, M.D., Ph.D.; Other
Eligibility: 18 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions: Myozyme® (alglucosidase alfa); Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 1 Month and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2020
U.S. locations: 4
States / cities: Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more

Conditions: Late-onset Pompe Disease
Interventions: BMN 701; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 8
States / cities: Phoenix, Arizona • Orange, California • Gainesville, Florida + 5 more

Conditions: Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
Interventions: Alglucosidase Alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2013
U.S. locations: 9
States / cities: Orange, California • Gainesville, Florida • Kansas City, Kansas + 6 more

Conditions: Pompe Disease Infantile-Onset
Interventions: ATB200, AT2221; Biological · Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 0 Years to 17 Years
U.S. locations: 2
States / cities: Oakland, California • Gainesville, Florida

Conditions: Glycogen Storage Disease Type II
Interventions: Alglucosidase alfa GZ419829; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 0 Days to 6 Months
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 5
States / cities: Hawthorne, New York • Durham, North Carolina • Cincinnati, Ohio + 2 more

Conditions: Pompe Disease (Late-onset)
Interventions: Clenbuterol, Placebos; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]); Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
U.S. locations: 74
States / cities: Sheffield, Alabama • Anchorage, Alaska • Phoenix, Arizona + 71 more

Conditions: Pompe Disease
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 110 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2021
U.S. locations: 6
States / cities: Phoenix, Arizona • Durham, North Carolina • Hershey, Pennsylvania + 3 more

Conditions: Glycogen Storage Disease Type II;Pompe's Disease
Interventions: Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 3 Years and older
Enrollment: 101 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2023
U.S. locations: 21
States / cities: Phoenix, Arizona • Los Angeles, California • Orange, California + 18 more

Conditions: Pompe Disease
Interventions: Muscle ultrasound, Electrical Impedance Myography; Device · Other
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 5
States / cities: Oakland, California • Orange, California • Omaha, Nebraska + 2 more

Conditions: Pompe Disease
Interventions: duvoglustat, rhGAA; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2013
U.S. locations: 11
States / cities: Phoenix, Arizona • Scottsdale, Arizona • Orange, California + 8 more

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 (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: 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: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions: SPK-3006; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2032
U.S. locations: 10
States / cities: Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more

Conditions: Glycogen Storage Disease Type II Pompe Disease
Interventions: Avalglucosidase Alfa; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2022
U.S. locations: 8
States / cities: Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 5 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: 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
Interventions: respiratory muscle strength training; Other
Lead sponsor: Duke University; Other
Eligibility: 3 Years to 99 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2012
U.S. locations: 1
States / cities: Durham, North Carolina

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

Extension Study for Patients Who Have Participated in a BMN 701 Study

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

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

Immune Tolerance Induction Study

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Alglucosidase Alfa Temporary Access Program

Alglucosidase Alfa Pompe Safety Sub-Registry

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Pompe Disease QMUS and EIM

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

Drug-drug Interaction Study

Genetic and Family Studies of Inherited Muscle Diseases

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

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Avalglucosidase Alfa Extension Study

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)

Prophylactic Ranibizumab for Exudative Age-related Macular Degeneration

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease