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A multicenter, randomized, placebo-controlled, double-blind phase 3 trial with open-arm comparison indicates safety and efficacy of nephroprotective therapy with ramipril in children with Alport syndrome

Kidney International Jan 26, 2020

Gross O, Tönshoff B, Weber LT, et al. - Given renal failure develops early in life in children with Alport syndrome, and because there exists uncertainty regarding the safety and efficacy of preemptive nephroprotective therapy, researchers analyzed pediatric patients treated with ramipril for three to six years and followed-up for six months in order to determine these parameters in this randomized, placebo-controlled, double-blind trial in 14 German sites. An open-arm control, comprising pretreated children and those whose parents rejected randomization, was contrasted with prospective real-world data from untreated children. No safety issues were found associated with ramipril therapy. Albeit not significant, the effectiveness of ramipril therapy was cautiously demonstrated by the findings: a reduced risk of disease progression by almost half, as well as the reduced slope of albuminuria progression and reduction in decline in glomerular filtration were noted in relation to ramipril treatment in the randomized arm. Therefore, it was proved that early initiation of therapy is safe and likely slow renal failure by many years, this implies that preemptive therapy is valuable. Therefore, the incorporation of genetic testing for Alport-associated gene-variants could be beneficial for screening programs for glomerular hematuria in children and young adults.
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