Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Condition: Fabry Disease
MeSH terms: Fabry Disease
Intervention: Migalastat HCl 150 mg
Intervention description: migalastat HCl 150 mg capsule
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 22.0
Sex: All
Minimum age: 12 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Lysosomal storage disease; migalastat; AT1001; Galafold

Summary:

      This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics
      (PD), and efficacy of migalastat treatment in pediatric participants 12 to <18 years of age
      with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.
    

Eligibility criteria:

        Key Inclusion Criteria

          -  Willing and able to provide written consent or assent (participant and parent/legal
             guardian, as applicable)

          -  Male or female between 12 and <18 years of age diagnosed with Fabry disease

          -  Confirmed, amenable GLA variant

          -  Participant weighed at least 45 kg (99 pounds) at screening

          -  Participant had never been treated with ERT or had not received ERT for 14 days prior
             to screening

          -  Participant had at least 1 complication (such as, laboratory abnormality and/or
             sign/symptom) of Fabry disease

          -  Participant was able to swallow study medication whole

        Key Exclusion Criteria

          -  Had moderate or severe renal impairment (estimated glomerular filtration rate (eGFR)
             <60 milliliter/minute/1.73 meter squared (m^2) at screening)

          -  Had advanced kidney disease requiring dialysis or kidney transplantation

          -  History of allergy or sensitivity to study medication (including excipients) or other
             iminosugars (for example, miglustat, miglitol)

          -  Had received any gene therapy at any time or anticipated starting gene therapy during
             the study period

          -  Required treatment with Glyset (miglitol) and/or Zavesca (miglustat) within 6 months
             before screening or throughout the study

          -  Required treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta)
             within 14 days before screening or throughout the study

          -  Participant was treated or had been treated with any investigational/experimental
             drug, biologic or device within 30 days before screening

          -  Any intercurrent illness or condition or concomitant medication use considered to be a
             contraindication at screening or baseline or that may have precluded the participant
             from fulfilling the protocol requirements or suggested to the investigator that the
             potential participant may have had an unacceptable risk by participating in this study

          -  Pregnant or breast-feeding or planned to become pregnant during the study period

          -  Otherwise unsuitable for the study in the opinion of the investigator