Table 4

Composite clinical outcome: number of patients (mITT population)

ComponentMigalastat (n=34)ERT (n=18)
Renal8 (24%)
↑Proteinuria (6), ↓GFR (2)
6 (33%)
↑Proteinuria (4), ↓GFR (3)
Cardiac2 (6%)
Chest pain, VT/chest pain
3 (17%)
Cardiac failure, dyspnoea, arrhythmia
CNS0 (0%)1 (6%)
Death0 (0%)0 (0%)
Any10 (29%)8 (44%)
  • Analyses undertaken in the mITT patients (randomised patients with amenable mutations receiving at least one dose of study drug and having baseline and postbaseline mGFRiohexol and eGFRCKD-EPI measures). Proteinuria event defined as >33% increase in 24-hour urine protein and level >300 mg; GFR event defined as >15 mL/min decline in eGFRCKD-EPI and level <90 mL/min/1.73 m2/year. Two patients in the ERT group experienced one cardiac and one renal event each.

  • CNS, central nervous system; eGFRCKD-EPI, estimated GFR using the Chronic Kidney Disease Epidemiology Collaboration formula; ERT, enzyme replacement therapy; GFR, glomerular filtration rate; mGFRiohexol, measured GFR using iohexol clearance; mITT, modified intention to treat; TIA, transient ischaemic attack; VT, ventricular tachycardia.