EARLY-PRO-TECT
Early Treatment of Children with Alport Syndrome
Doente / População Intervenção / Exposição Comparação Desfecho
In 80 children with Alport syndrome, early ramipril delayed progression of albuminuria compared with placebo.
N
66
Desenho
Phase 3 RCT in paediatric patients
Desfecho
Co-primary: safety (adverse drug reactions) and efficacy (time to disease progression)
Relevância
3
ResultadoRamipril reduced disease-progression risk by ~half but not significantly (randomized arm HR 0.51, 95% CI 0.12–2.20; Bayesian synthesis with open-arm data HR 0.52, 95% CI 0.19–1.39); safe (AE rate-ratio 1.00, 95% CI 0.66–1.53).
Gross O, et al. Kidney Int. 2020;97(6):1275-1286. 10.1016/j.kint.2019.12.015
Discussão e crítica
Rare-disease trials are necessarily small; supports early RAS blockade in Alport syndrome. Bardoxolone (CARDINAL) later failed to gain approval.