Trial Entry Criteria

Consult with a healthcare professional to see if you meet the entry criteria.

How do I qualify?

Inclusion Criteria:

  • Male and female subjects between 18 and 65 years of age, inclusive
  • Diagnosis of Pompe disease

Enzyme Replacement Therapy (ERT)-experienced subjects (ambulatory):

  • Received ERT with alglucosidase alfa for the previous 2-6 years, inclusive
  • Currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
  • Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
  • Upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

ERT-experienced subjects (non-ambulatory):

  • Received ERT with alglucosidase alfa (Myozyme/Lumizyme) for ≥2 years
  • Wheelchair-bound

ERT-Naïve subjects (ambulatory):

  • Must be able to walk 200-500 meters on the 6MWT
  • Upright FVC must be 30% to 80% of predicted normal value
  • Never received alglucosidase alfa

Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):

  • Has received ERT with alglucosidase alfa for >7years, inclusive
  • Currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
  • Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
  • Upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

Exclusion Criteria:

  • Received treatment with prohibited medications within 30 days of Baseline Visit
  • If female, is pregnant or breastfeeding at screening
  • Whether male or female, planning to conceive a child during the study
  • Has a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
  • Has a history of allergy or sensitivity to miglustat or other iminosugars
  • With active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis. All subjects with autoimmune disease must be discussed with the Amicus Medical Monitor
  • With active bronchial asthma. All subjects with bronchial asthma must be discussed with the Amicus Medical Monitor