Search | Clinical Trials Finder

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: 4
States / cities: Irvine, California • St Louis, Missouri • Durham, North Carolina + 1 more

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, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions: Neutralizing Antibody to SPK-3006 capsid; Diagnostic Test
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 6
States / cities: Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more

Conditions: Glycogen Storage Disease Type II, Pompe Disease
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 2,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2034
U.S. locations: 92
States / cities: Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 70 more

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: LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
Interventions: Not listed
Lead sponsor: Virginia Commonwealth University; Other
Eligibility: 6 Years to 50 Years
Enrollment: 1,000 participants
Timeline: 2023 – 2029
U.S. locations: 1
States / cities: Richmond, Virginia

Conditions: Pompe Disease (Late-onset)
Interventions: zocaglusagene nuzaparvovec; Genetic
Lead sponsor: Astellas Gene Therapies; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2035
U.S. locations: 3
States / cities: Orange, California • Palo Alto, California • Salt Lake City, Utah

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 (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: Pompe Disease, Muscle Weakness
Interventions: Resistance Exercise Training; Other
Lead sponsor: University of California, Irvine; Other
Eligibility: 16 Years to 75 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: Irvine, California

Conditions: Glycogen Storage Disease Type II
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 6
States / cities: Birmingham, Alabama • Gainesville, Florida • Miami, Florida + 3 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 (Late-onset)
Interventions: Cipaglucosidase Alfa, Miglustat; Biological · Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 0 Years to 17 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 9
States / cities: Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more

Conditions: Pompe Disease, Pompe Disease (Late-onset), GAA Deficiency
Interventions: Observational; Other
Lead sponsor: Duke University; Other
Eligibility: 3 Months to 54 Months
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Late-onset Pompe Disease
Interventions: Not listed
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 17
States / cities: Irvine, California • Gainesville, Florida • Decatur, Georgia + 14 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

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: 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)
Interventions: No Intervention; Other
Lead sponsor: Astellas Gene Therapies; Industry
Eligibility: 16 Years to 69 Years
Enrollment: 119 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 10
States / cities: Atlanta, Georgia • Kansas City, Kansas • Ann Arbor, Michigan + 7 more

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: Glycogen Storage Disease Type II, Glycogenosis 2
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 8
States / cities: Aurora, Colorado • Wichita, Kansas • Cambridge, Massachusetts + 5 more

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: 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

Search by objective public record fields.

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

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

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Pompe Disease Registry Protocol

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

Long-Term Development of Muscular Dystrophy Outcome Assessments

Gene Transfer Study in Patients With Late Onset Pompe Disease

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

High Dose Inspiratory Muscle Training in LOPD

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

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

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

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

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

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Alglucosidase Alfa Temporary Access Program

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease

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

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