Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
Condition: Fabry Disease
MeSH terms: Fabry Disease
Intervention: Migalastat HCl; Agalsidase Beta; Agalsidase Alfa
Intervention description: Oral capsules, single dose; IV infusion, single dose; IV infusion, single dose
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Sequential Assignment
Intervention model description: A 2-stage design was used to study the effects of 2 dose levels of migalastat, while a 2- or 3-period design within each stage enabled study of the effects of one drug on the PK, pharmacodynamics, and safety of the other drug.
Masking: None (Open Label)
Enrollment: 20.0
Sex: Male
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: Amicus Therapeutics; AT1001; Galafold; Migalastat; Pharmacokinetics

Summary:

      The objective was to determine the effects of a single dose of migalastat hydrochloride (HCl)
      (migalastat) 150 and 450 milligrams (mg) on the safety and plasma pharmacokinetics (PK) of
      agalsidase and the effects of agalsidase on the safety and PK of migalastat 150 mg.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male diagnosed with Fabry disease and between 18 and 65 years of age, inclusive

          -  Body mass index between 18-35 kg per meter squared

          -  Had initiated treatment with agalsidase at least 1 month prior to screening, and had
             received at least 2 infusions before screening

          -  Had stable dose level, dosing regimen, and form of agalsidase for at least 1 month
             before screening

          -  Had an estimated creatinine clearance greater than or equal to 50 milliliters
             (mL)/minute at screening

          -  Agreed to use medically accepted methods of contraception during the study and for 30
             days after study completion

          -  Were willing and able to provide written informed consent

        Exclusion Criteria:

          -  Had a documented transient ischemic attack, ischemic stroke, unstable angina, or
             myocardial infarction within the 3 months before screening

          -  Had 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)

          -  History of allergy or sensitivity to study drug (including excipients) or other
             iminosugars (such as miglustat, miglitol)

          -  Required a concomitant medication prohibited by the protocol: Glyset® (miglitol), or
             Zavesca® (miglustat)

          -  Any investigational/experimental drug or device within 30 days of screening, except
             for use of investigational enzyme replacement therapy for Fabry disease

          -  Had any intercurrent illness or condition that might have precluded the participant
             from fulfilling the protocol requirements or suggested to the investigator that the
             potential participant might have had an unacceptable risk by participating in this
             study