A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Completed · Phase 3 · Has a placebo group

Conditions studied: Pompe Disease (Late-onset)

In brief

This is a phase 3 double-blind randomized study to study the efficacy and safety of intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe Disease compared with Alglucosidase Alfa/placebo.

Key facts

Study ID

NCT03729362

Run by

Amicus Therapeutics

People needed

125

Starts

2018-12-04

Expected to finish

2021-01-15

Last updated by the study team

2025-09-11

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subject must provide signed informed consent prior to any study-related procedures being performed.
• Male and female subjects are ≥ 18 years old and weigh ≥ 40 kg at screening.
• Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug.
• Subject must have a diagnosis of LOPD based on documentation of one of the following:
• deficiency of GAA enzyme
• GAA genotyping
• Subject is classified as one of the following with respect to ERT status:
• ERT-experienced, defined as currently receiving standard of care ERT (alglucosidase alfa) at the recommended dose and regimen (ie, 20 mg/kg dose every 2 weeks) for ≥ 24 months
• ERT-naïve, defined as never having received investigational or commercially available ERT
• Subject has a sitting FVC ≥ 30% of the predicted value for healthy adults (National Health and Nutrition Examination Survey III) at screening.
• Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria:
• both screening values of 6MWD are ≥ 75 meters
• both screening values of 6MWD are ≤ 90% of the predicted value for healthy adults
• the lower value of 6MWD is within 20% of the higher value of 6MWD

You may not qualify if…

• Subject has received any investigational therapy or pharmacological treatment for Pompe disease, other than alglucosidase alfa, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before Day 1 or is anticipated to do so during the study.
• Subject has received gene therapy for Pompe disease
• Subject is taking any of the following prohibited medications within 30 days before Day 1:
• miglitol (eg, Glyset)
• miglustat (eg, Zavesca)
• acarbose (eg, Precose or Glucobay)
• voglibose (eg, Volix, Vocarb, or Volibo)
• Note: None of these medications have a half-life that, when multiplied by 5, is longer than 30 days.
• Subject requires the use of invasive or noninvasive ventilation support for > 6 hours per day while awake.
• Subject has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221.
• Subject has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms.
• Subject, if female, is pregnant or breastfeeding at screening.
• Subject, whether male or female, is planning to conceive a child during the study.
• Subject does not have documentation of diagnosis of Pompe disease and refuses to undergo genetic testing.

Where it is running

• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States
• University of California, Irvine — Irvine, California, United States
• UF Helath: University of Florida Clinical Research Center — Gainesville, Florida, United States
• University of South Florida Research Center — Tampa, Florida, United States
• Emory Clinic — Atlanta, Georgia, United States
• Indiana University Health Neuroscience Center — Indianapolis, Indiana, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• University of Minnesota Clinical Research Unit — Minneapolis, Minnesota, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• Billings Clinic — Billings, Montana, United States
• Hackensack University Medical Center — Hackensack, New Jersey, United States
• The Feinstein Institute for Medical Research — Manhasset, New York, United States
• NYU School of Medicine — New York, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• University of Cincinnati Neurology — Cincinnati, Ohio, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• The Ohio State University Wexner Medical Center — Columbus, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Penn State Health Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States
• University of Texas Health Science Center San Antonio — San Antonio, Texas, United States
• University of Utah, Center for Clinical and Translational Sciences — Salt Lake City, Utah, United States
• Lysosomal and Rare Disorders Research — Fairfax, Virginia, United States
• Neuromuscular Research Center — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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