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

Amicus Therapeutics16 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fabry Disease

Interventions

migalastat HCl 150 mgDRUG

migalastat HCl 150 mg capsule

Eligibility

Sex: ALLMin age: 12 YearsMax age: 17 Years

Medical Language ↔ Plain English

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 * If of reproductive potential, both male and female subjects agreed 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: * Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was \< 60 mL/min/1.73 m2 * Subject had advanced kidney disease requiring dialysis or kidney transplantation * Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat * Subject anticipated starting gene therapy during the study period * Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study * Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol) * Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta) * Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat) * Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period * In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements

Locations (6)

University of South Florida

Tampa, Florida, United States

The Emory Clinic

Atlanta, Georgia, United States

University of Minnesota Masonic Children's Hospital and Clinics

Minneapolis, Minnesota, United States

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug

Number of subjects with TEAE, SAE, and AE leading to discontinuation during the study period

Time frame: Entire study

Secondary Outcomes

Change From Baseline to Month 24 in Estimated Glomerular Filtration Rate (eGFR)

Estimated GFR was calculated using the modified Schwartz formula according to the standards of the central laboratory.

Time frame: Baseline, Month 24

Change From Baseline to Month 24 in Urine Protein Levels

Renal function was assessed by urine protein levels (mg/L). Urine samples were collected as part of urinalysis.

Time frame: Baseline, Month 24

Change From Baseline to Month 24 in Urine Albumin

Renal function was assessed by urine albumin levels (mg/L). Urine samples were collected as part of urinalysis

Time frame: Baseline, Month 24

Change From Baseline to Month 24 in Left Ventricular Mass Index (LVMi)

LVMi was assessed as a measure of cardiac impairment in the study participants. LVMi values for both M-mode and 2D views are presented.

Time frame: Baseline, Month 24

Change From Baseline to Month 24 in Pediatric and Quality of Life Inventory™ (PedsQL™) Scores

The Pediatric Quality of Life Inventory (PedsQL™) was a modular approach to measuring health-related quality of life (QoL) in healthy children and adolescents and those with acute and chronic health conditions. All components of the PedsQL were scored based on a scale of 0 (never) to 4 (almost always) and linearly transformed to a 0 to 100 scale as follows: 0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0. Psychosocial, physical, and total scores were calculated based on the response to the questions within the patient reported outcome. The psychosocial score for the PedsQL encompassed 15 questions relating to the subjects' feelings, social interaction with others, and school. The physical score was derived from answers to 8 questions about the subjects' ease of managing physical activity. Total scores were the sum of all the item scores over the number of items answered on all the scales. Change from baseline values of \<0 represents worsening, 0 equals no change, and \>0 represents improvement.

Data from ClinicalTrials.gov

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