20
Participants
Start Date
March 31, 2016
Primary Completion Date
December 31, 2017
Study Completion Date
February 28, 2018
Rimeporide
"Cohort 1:~50 mg TID in patients with a body weight ≤ 30kg at Baseline and 75 mg TID in patients with a body weight \> 30kg at Baseline~Cohort 2:~100mg TID in patients with a body weight ≤ 30kg at baseline and 150 mg TID in patients with a body weight \> 30kg at Baseline~Cohort 3:~150 mg TID in patients with a body weight ≤ 30kg at baseline and 200 mg TID in patients with a body weight \> 30kg at Baseline~Cohort 4:~200 mg TID in patients with a body weight ≤ 30kg at Baseline and 300 mg TID mg TID in patients with a body weight \> 30kg at Baseline"
San Raffaele Hospital, Milan
I-Motion - Hôpital Armand Trousseau, Paris
Santa Creu i Sant Pau Hospital, Barcelona
UCL Institute of Child Health and Great Ormond Street Hospital, London
EspeRare Foundation
OTHER