Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04049760
• Other study ID #: AT1001-036
• Secondary ID: 2019-000222-21
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: October 14, 2019
• Est. completion date: December 1, 2025

Study information

• Verified date: January 2024
• Source: Amicus Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 15
• Est. completion date: December 1, 2025
• Est. primary completion date: December 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 17 Years

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• migalastat HCl 150 mg
migalastat HCl 150 mg capsule

Locations

Country	Name	City	State
United Kingdom	Royal Free London NHS Foundation Trust	London
United States	The Emory Clinic	Atlanta	Georgia
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	Lysosomal & Rare Disorders Research & Treatment Center	Fairfax	Virginia
United States	University of Minnesota Masonic Children's Hospital and Clinics	Minneapolis	Minnesota
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug		Month 60
Primary	change in body weight in kilograms		baseline over time; Up to 5 years
Primary	change in height in centimeters		baseline over time; Up to 5 years
Primary	changes in Electrocardiogram (ECG) results	A 12-lead ECG will be obtained.	baseline over time; Up to 5 years
Primary	incidence of changes in echocardiogram results	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.	baseline over time; Up to 5 years
Primary	change in Tanner stage	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.	Every 6 Months; Up to 5 years
Primary	incidence of concomitant medications use		Every 1 Month; Up to 5 years
Secondary	change in plasma levels of lyso-Gb3		Every 6 Months; Up to 5 years
Secondary	change in eGFR		Every 6 Months; Up to 5 years
Secondary	change in urine protein		Every 6 Months; Up to 5 years
Secondary	change in albumin levels		Every 6 Months; Up to 5 years
Secondary	change in Left Ventricular Mass Index (LVMi)		Every Year; Up to 5 years
Secondary	change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores	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.	Every 3 months; Up to 5 years
Secondary	change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores	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.	Every 3 months; Up to 5 years