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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: 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: Albuterol, Placebo; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Late-Onset Pompe Disease, Lysosomal Disease
Interventions: IMT therapy using the Pr02 mobile device; Device
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease
Interventions: Observational; Other
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2029
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Late-onset Pompe Disease
Interventions: DNL952; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 3
States / cities: Irvine, California • Durham, North Carolina • Fairfax, Virginia

Conditions: Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 6 Months to 36 Months
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 3
States / cities: Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio

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: Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Interventions: Observational study; Other
Lead sponsor: Duke University; Other
Eligibility: 12 Years and older
Enrollment: 73 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease
Interventions: BMN 701; Biological
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 13 Years and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2013
U.S. locations: 3
States / cities: La Jolla, California • Gainesville, Florida • Kansas City, Kansas

Conditions: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions: Aldurazyme (laronidase); Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2032
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Lysosomal Storage Diseases
Interventions: Enzyme assay and molecular sequencing; Diagnostic Test
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Fairfax, Virginia

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
Interventions: Not listed
Lead sponsor: O & O Alpan LLC; Other
Eligibility: 18 Years and older · Female only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Pompe Disease
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 8 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005
U.S. locations: 5
States / cities: Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more

Conditions: Pompe Disease (Late-onset)
Interventions: AT2221, ATB200; Drug · Biological
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 119 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 18
States / cities: Little Rock, Arkansas • Irvine, California • Gainesville, Florida + 14 more

Conditions: Pompe Disease
Interventions: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase, Rapamycin, saline, Rituxan, Diphenhydramine, Acetaminophen, Lidocaine, LMX 4 Topical Cream; Genetic · Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 50 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: Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated; Biological · Drug · Other
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: Not listed
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2034
U.S. locations: 15
States / cities: Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 6 Months and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2010
U.S. locations: 9
States / cities: Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 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: 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

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: Pompe Disease, Glycogen Storage Disease Type II
Interventions: Observation; Other
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: Not listed
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 4
States / cities: Gainesville, Florida • St Louis, Missouri • Lake Success, New York + 1 more

Conditions: Glycogen Storage Disease
Interventions: Lingual Muscle Training; Other
Lead sponsor: Duke University; Other
Eligibility: 12 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Durham, North Carolina

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Avalglucosidase Alfa Extension Study

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

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

High Dose Inspiratory Muscle Training in LOPD

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Identification of Tongue Involvement in Late-Onset Pompe Disease

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

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

Pregnancy and Birth Outcome in Women With Pompe Disease

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

A Global Prospective Observational Registry of Patients With Pompe Disease

High Dose or High Dose Frequency Study of Alglucosidase Alfa

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

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

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)