Migalastat HCl 20 mg: Migalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.
Study summary
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
Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
Inclusion Criteria
* 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.
* Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease
* If of reproductive potential, both male and female subjects agree to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat.
Exclusion Criteria
* 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).
* Has received any gene therapy at any time or anticipates starting gene therapy during the study period.
* Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.
* Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
* Pregnant or breastfeeding
* Otherwise unsuitable for the study in the opinion of the investigator
Primary outcome measure(s)
Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug — Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose)
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Migalastat — 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
Pharmacokinetics (PK): Minimum Observed Plasma Concentration (Cmin) of Migalastat — 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) of Migalastat — 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
Trial sites (12)
Facility
City
Region
Status
Emory Genetics
Atlanta
Georgia
Recruiting
University of Minnesota Masonic Children's Hospital
Minneapolis
Minnesota
Recruiting
Atrium Health Levine Children's Hospital
Charlotte
North Carolina
Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati
Ohio
Recruiting
UPMC Children's Hospital of Pittsburgh
Pittsburgh
Pennsylvania
Recruiting
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax
Virginia
Recruiting
Universitair Ziekenhuis (UZ) Leuven
Leuven
Vlaams-Brabant
Recruiting
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
Münster
North Rhine-Westphalia
Recruiting
Kitasato University Hospital
Sagamihara
Kanagawa
Not Yet Recruiting
Hospital Universitario de la Paz
Madrid
Madrid
Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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