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

• Register: ClinicalTrials.gov
• Last refreshed on: 22 January 2024
• Main ID: NCT04049760
• Date of registration: 30/07/2019
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
• Scientific 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
• Date of first enrolment: October 14, 2019
• Target sample size: 15
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04049760
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Spain, United Kingdom, United States

Key inclusion & exclusion criteria

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



Age minimum: 12 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: migalastat HCl 150 mg

Primary Outcome(s)

incidence of changes in echocardiogram results [Time Frame: baseline over time; Up to 5 years]

change in height in centimeters [Time Frame: baseline over time; Up to 5 years]

change in body weight in kilograms [Time Frame: baseline over time; Up to 5 years]

incidence of concomitant medications use [Time Frame: Every 1 Month; Up to 5 years]

change in Tanner stage [Time Frame: Every 6 Months; Up to 5 years]

changes in Electrocardiogram (ECG) results [Time Frame: 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 [Time Frame: Month 60]

Secondary Outcome(s)

change in eGFR [Time Frame: Every 6 Months; Up to 5 years]

change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores [Time Frame: Every 3 months; Up to 5 years]

change in albumin levels [Time Frame: Every 6 Months; Up to 5 years]

change in urine protein [Time Frame: Every 6 Months; Up to 5 years]

change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores [Time Frame: Every 3 months; Up to 5 years]

change in Left Ventricular Mass Index (LVMi) [Time Frame: Every Year; Up to 5 years]

change in plasma levels of lyso-Gb3 [Time Frame: Every 6 Months; Up to 5 years]

Secondary ID(s)

AT1001-036

2019-000222-21

Source(s) of Monetary Support

Please refer to primary and secondary sponsors