Intervention: Miglustat
Trial phase: Phase 3
ATC code: A16AX06
SMILES: CCCCN1C[C@H](O)[C@@H](O)[C@@H](O)[C@H]1CO
Sex: All
Age groups: Adult | Older Adult
Healthy volunteers: No
Participants at risk: 85
Group description: All participants who received at least 1 dose of study drug (cipaglucosidase alfa/miglustat).
Eligibility criteria:
Inclusion Criteria:

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

Exclusion Criteria

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.