Drug-drug Interaction Study
Condition: Pompe Disease
MeSH terms: Glycogen Storage Disease Type II
Intervention: duvoglustat; rhGAA
Intervention description: Single oral dose; Single intravenous infusion
Intervention MeSH terms: 1-Deoxynojirimycin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 25.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: Amicus Therapeutics; duvoglustat; AT2220; alpha-glucosidase; alglucosidase alfa

Summary:

      This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant
      human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe
      Disease.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female, diagnosed with Pompe disease and between 18 and 65 years of age,
             inclusive

          -  Participant has been on a stable regimen and dose of rhGAA for at least 3 months
             before screening (stable regimen defined as currently receiving rhGAA every 2 weeks
             and stable dose defined as not varying by more than ± 10%)

          -  Participant has an estimated glomerular filtration rate (eGFR) ≥ 50 mL/min at
             Screening; eGFR to be estimated using the 4-parameter Modification of Diet in Renal
             Disease (MDRD) equation:

        eGFR (mL/min/1.73 m^2) = 175 x (Scr)^(-1.154) x (Age)^(-0.203) x (0.742 if female) x (1.212
        if African-American)

          -  Male and female participants of childbearing potential agree to use medically accepted
             methods of contraception during the study and for 30 days after study completion

          -  Participant is willing and able to provide written informed consent and is able to
             comply with all study procedures

        Exclusion Criteria:

          -  Participant has had a documented transient ischemic attack, ischemic stroke, unstable
             angina, or myocardial infarction within the 3 months before Screening

          -  Participant has clinically significant unstable cardiac disease (for example, cardiac
             disease requiring active management, such as symptomatic arrhythmia, unstable angina,
             or New York Heart Association class III or IV congestive heart failure)

          -  Participant requiring mechanical ventilation or is confined to a wheelchair

          -  Participant has a history of allergy or sensitivity to study drug (including
             excipients) or other iminosugars (for example, miglustat, miglitol)

          -  Participant is pregnant or breastfeeding

          -  Participant tests positive for hepatitis B surface antigen or hepatitis C antibody

          -  Participant has received any investigational/experimental drug or device within 30
             days of Screening

          -  Participant has any intercurrent illness or condition that may preclude the
             participant from fulfilling the protocol requirements or suggests to the investigator
             that the potential participant may have an unacceptable risk by participating in this
             study