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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: 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: Pompe Disease, Glycogen Storage Disease II
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: 6 Years to 18 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2021
U.S. locations: 1
States / cities: Durham, North Carolina

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: 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
Interventions: Not listed
Lead sponsor: University of California, Irvine; Other
Eligibility: 8 Years and older
Enrollment: 921 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Irvine, California

Conditions: Pompe Disease
Interventions: ACTUS-101; Genetic
Lead sponsor: AskBio Inc; Industry
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Durham, North Carolina

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: 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: 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: VAL-1221, RhGAA; Drug
Lead sponsor: Valerion Therapeutics, LLC; Industry
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 2
States / cities: Orange, California • Durham, North Carolina

Conditions: Pompe Disease, Pregnancy
Interventions: avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666); Biological
Lead sponsor: Sanofi; Industry
Eligibility: Female only
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2032
U.S. locations: 1
States / cities: Bridgewater, New Jersey

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: 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: 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
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: 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, 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: 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: 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: Glycogen Storage Disease, Pompe Disease
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Female only
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2024
U.S. locations: 2
States / cities: Cambridge, Massachusetts • Grand Rapids, Michigan

Conditions: Healthy, Late Onset Pompe Disease
Interventions: ABX1100 injection for IV infusion, Placebo injection for IV infusion; Drug
Lead sponsor: Aro Biotherapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 3
States / cities: Orange, California • St Louis, Missouri • 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: Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
Interventions: Not listed
Lead sponsor: O & O Alpan LLC; Other
Eligibility: 1 Day to 100 Years
Enrollment: 20,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Fairfax, Virginia

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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)

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

Pompe Telemedicine Developmental Study

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

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

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Gene Transfer Study in Patients With Late Onset Pompe Disease

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

High Dose Inspiratory Muscle Training in LOPD

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Avalglucosidase Alfa Pregnancy Study

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

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

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

A Global Prospective Observational Registry of Patients With Pompe Disease

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

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

Pregnancy and Birth Outcome in Women With Pompe Disease

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

Pompe Lactation Sub-Registry

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

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

Investigating Lysosomal Storage Diseases in Minority Groups