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