Efficacy and Safety Study to Delay Renal Failure in Children With Alport Syndrome
Status:
Completed
Trial end date:
2019-03-01
Target enrollment:
Participant gender:
Summary
This is a phase III, multi-centre, randomised, placebo-controlled, patient and
investigator-blind study in paediatric patients with early stages of Alport syndrome to
assess the safety and efficacy of the ACEi ramipril in slowing disease progression.
Alport syndrome stages that describe the extent of renal damage and loss of function are
defined as:
- 0 Microhaematuria without microalbuminuria (usually at birth)
- I Microalbuminuria (30-300 mg albumin/gCrea)
- II Proteinuria >300 mg albumin/gCrea
- III > 25% decline of normal renal function (creatinine clearance)
- IV End stage renal failure (ESRF)
Eligible patients with Alport stages 0 and I will be randomly assigned at a 2:1 ratio to
receive once daily ramipril or placebo. In addition, Alport stage II patients may be treated
open Label. Eligible patients who, or whose parents/legal guardian refuse randomisation after
eligibility is confirmed, and patients who have been treated with ramipril prior to the
study, may be treated open-label with ramipril as per protocol. The total number of patients
will not exceed 120, with the number of randomised patients not exceeding 60, and the number
of patients treated open label from Day 1 of the study aimed to be approximately 60.
Randomised patients whose disease progresses to the next disease level during the 3 year
treatment period will be unblinded, and open label ramipril treatment will be initiated and
continued, respectively, depending on prior treatment randomisation.
Phase:
Phase 3
Details
Lead Sponsor:
Institut fuer anwendungsorientierte Forschung und klinische Studien GmbH
Collaborators:
German Federal Ministry of Education and Research University Medical Center Goettingen