A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
ConditionFabry Disease
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-11
SponsorAmicus Therapeutics
About this trial
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
Eligibility criteria
Qualifiers
Male or female subjects, diagnosed with Fabry disease who are between ages 2 and < 12 years at randomization (subjects aged 11 years must have birthdays > 30 days after randomization)
Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable.
Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
Subject has not received ERT (eg, Replagal® [agalsidase alfa] or Fabrazyme® [agalsidase beta]) for at least 14 days prior to Baseline visit.
Disqualifiers
Has moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at Visit 1 [screening]).
Has advanced kidney disease requiring dialysis or kidney transplantation.
History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).
Trial design
Treatments tested in this trial
- Migalastat HCl 20 mg
Treatment groups
8 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov