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 groupLocations
11Belgium
Universitair Ziekenhuis (UZ) Leuven3000, LeuvenVlaams-Brabant, Belgium
Germany
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie48149, MünsterNorth Rhine-Westphalia, Germany
Spain
Hospital Universitario de la Paz28046, MadridMadrid, Spain
United Kingdom
Great Ormond Street Hospital for Children NHS Foundation TrustWC1N 3JH, London United Kingdom
Sponsors and collaborators
Amicus Therapeutics
Lead sponsor
Source ClinicalTrials.gov