Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) with Fabry Disease
- Registration Number
- NCT04049760
- Lead Sponsor
- Amicus Therapeutics
- Brief Summary
This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects \> 12 Years of Age With Fabry Disease and Amenable GLA Variants
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 15
- Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
- 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'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
- Has moderate or severe renal impairment (eGFR <60 ml/min/1.73 m2 at screening)
- Has advanced kidney disease requiring dialysis or kidney transplantation
- History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)
- Has received any gene therapy at any time or anticipates starting gene therapy during the study period
- Requires treatment with Glyset (miglitol), Zavesca (miglustat) within 6 months before screening or throughout the study
- Requires treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study
- Subject is treated or has 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 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 breast-feeding
- Otherwise unsuitable for the study in the opinion of the investigator
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description migalastat HCl 150 mg migalastat HCl 150 mg One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.
- Primary Outcome Measures
Name Time Method change in body weight in kilograms baseline over time; Up to 5 years incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug Month 60 changes in Electrocardiogram (ECG) results baseline over time; Up to 5 years A 12-lead ECG will be obtained.
incidence of changes in echocardiogram results baseline over time; Up to 5 years Systolic and diastolic heart function and structure is assessed by ultrasound of the heart. Echocardiogram parameters include left ventricular mass index (LVMi), ejection fraction, fractional shortening, left ventricular internal diameter end diastole and end systole, midwall fractional shortening, and wall thickness.
change in height in centimeters baseline over time; Up to 5 years change in Tanner stage Every 6 Months; Up to 5 years Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.
incidence of concomitant medications use Every 1 Month; Up to 5 years
- Secondary Outcome Measures
Name Time Method change in plasma levels of lyso-Gb3 Every 6 Months; Up to 5 years change in eGFR Every 6 Months; Up to 5 years change in urine protein Every 6 Months; Up to 5 years change in albumin levels Every 6 Months; Up to 5 years change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores Every 3 months; Up to 5 years The FPHPQ includes questions about Fabry disease-specific symptoms (eg, sweating, pain, dizziness and tiredness, heat and cold intolerance, swollen eyelids, gastrointestinal symptoms, feeling thirsty, difficulty hearing, ringing or buzzing noise in the ears, and ability and enjoyment to participate in sports). The frequency of these symptoms will be rated using a 5-point Likert scale (always (worse), often, sometimes, seldom, never (better)). Pain intensity is measured on a 10-point scale, numeric responses are given for onset of pain and school days missed, and yes/no questions are posed about difficulty hearing and other problems not specifically mentioned. There are 2 age-specific self-report versions for children 8 to 12 years and 13 to 18 years, respectively.
change in Left Ventricular Mass Index (LVMi) Every Year; Up to 5 years change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores Every 3 months; Up to 5 years The PedsQL™ is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. It consists of 23 items and includes questions about physical functioning, emotional functioning, social functioning, and school functioning relative to the prior 7 days, using a 5-point scale (never (better), almost never, sometimes, often and almost always (worse)). Both parents or legally-authorized representatives and subjects complete the appropriate version of the PedsQL independently of one another. Parents or legally-authorized representatives and subjects may self-administer the questions after introductory instructions are given by study site personnel.
Trial Locations
- Locations (6)
The Emory Clinic
🇺🇸Atlanta, Georgia, United States
University of Minnesota Masonic Children's Hospital and Clinics
🇺🇸Minneapolis, Minnesota, United States
Lysosomal & Rare Disorders Research & Treatment Center
🇺🇸Fairfax, Virginia, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Royal Free London NHS Foundation Trust
🇬🇧London, United Kingdom