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RECRUITINGPhase 3INTERVENTIONAL

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

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.

Who May Be Eligible (Plain English)

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 willing to sign a consent form 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DRUG

Migalastat HCl 20 mg

Migalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.

Locations (9)

Emory Genetics
Atlanta, Georgia, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, United States
Universitair Ziekenhuis (UZ) Leuven
Leuven, Vlaams-Brabant, Belgium
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
Münster, North Rhine-Westphalia, Germany
Hospital Universitario de la Paz
Madrid, Madrid, Spain
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
Manchester University NHS Foundation Trust
Manchester, United Kingdom