Trial Entry Criteria

Rallying towards a better future for Pompe Disease

How do I qualify? Study participant must meet all of the inclusion criteria and none of the exclusion criteria to be eligible to participate in the study.

Inclusion Criteria:

  • Study participant must provide signed informed consent prior to any study-related procedures being performed.
  • Male and female participants are ≥ 18 years old and weigh ≥ 40 kg at screening.
  • Female volunteers of childbearing potential and male volunteers must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug.
  • Study participant must have a diagnosis of Late Onset Pompe Disease (LOPD) based on documentation of one of the following:
    • deficiency of acid alpha glucosidase (GAA) enzyme
    • GAA genotyping
  • Study participant is classified as one of the following with respect to Enzyme Replacement Therapy (ERT) status:
    • ERT-experienced, defined as currently receiving standard of care ERT (alglucosidase alfa) at the recommended dose and regimen (i.e., 20 mg/kg dose every 2 weeks) for ≥ 24 months
    • ERT-naïve, defined as never having received investigational or commercially available ERT
  • Study participant has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy adults (National Health and Nutrition Examination Survey III) at screening.
  • Study participant performs two six-minute walk tests (6MWTs) at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria:
    • both screening values of six-minute walk distance (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

Exclusion Criteria:

  • Study participant 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.
  • Study participant has received gene therapy for Pompe disease.
  • Study participant is taking any of the following prohibited medications within 30 days before Day 1:1
    • miglitol (e.g. Glyset®)
    • miglustat (e.g. Zavesca®)
    • acarbose (e.g. Precose® or Glucobay®)
    • voglibose (e.g. Volix®, Vocarb®, or Volibo®)
  • Study participant 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.
  • Study participant 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 study participant 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.
  • Study participant, if female, is pregnant or breastfeeding at screening.
  • Study participant, 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.


  1. None of these medications have a half-life that, when multiplied by 5, is longer than 30 days.


  • Glyset - US: Pfizer Inc.
  • Zavesca - Actelion Pharmaceuticals Ltd.
  • Precose - US and several countries: Bayer Healthcare Pharmaceuticals Inc.; India: M/S. Jenburki Pharmaceuticals Ltd.
  • Glucobay - Bayer Intellectual Property GmbH
  • Volix - No US registration for voglibose; Outside US, trademark in India from Sun Pharma Laboratories, Ltd.
  • Vocarb - No US registration for vocarb; Outside US, trademark (application pending) in India from Glenmark Pharmaceuticals Ltd.
  • Volibo - No US registration for this mark; Outside US, trademark in India from Sun Pharma Laboratories, Ltd.