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

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

Time frame · Entire study

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

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

Time frame · Baseline, Month 24

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

Change From Baseline to Month 24 in Urine Protein Levels

Time frame · Baseline, Month 24

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

Change From Baseline to Month 24 in Urine Albumin

Time frame · Baseline, Month 24

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

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

Time frame · Baseline, Month 24

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

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

Time frame · Baseline, Month 24

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.

Change From Baseline to Month 24 in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ) Score for Pain Intensity

Time frame · Baseline, Month 24

The Fabry-specific Pediatric Health and Pain Questionnaire (FPHPQ) included questions about Fabry disease-specific symptoms. The assessment of "How bad is your pain today?" was measured on a 10- point scale from 0 (no pain) to 10 (pain as bad as you can imagine). A decrease from baseline indicates an improvement in the condition.

Number of Subjects Who Experienced Sudden Onset of Pain As Assessed Using the Fabry-Specific Health and Pain Questionnaire (FPHPQ)

Time frame · Month 24

Subjects were asked "In the last 3 months how many times did you experience sudden onset of pain?" and responses were reported in the FPHPQ. Responses were categorized as 0, 1 to 3, 4 to 6, and \> 6 occurrences of sudden onset of pain.

Change From Baseline to Month 24 in Plasma Levels of Lyso-Gb3

Time frame · Baseline, Month 24

Blood samples were collected for measurement of lyso-Gb3 levels in plasma. Plasma levels of lyso-Gb3 were measured using a validated liquid chromatography-mass spectrometry assay.