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Early Prospective Therapy Trial to Delay Renal Failure in Children With Alport Syndrome
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.
Age
2 - 18 years
Sex
ALL
Healthy Volunteers
No
University Medical Center Goettingen
Göttingen, Germany
Start Date
March 1, 2012
Primary Completion Date
September 1, 2018
Completion Date
March 1, 2019
Last Updated
June 17, 2020
66
ACTUAL participants
Ramipril
DRUG
placebo to ramipril
DRUG
Ramipril
DRUG
Lead Sponsor
Institut fuer anwendungsorientierte Forschung und klinische Studien GmbH
Collaborators
NCT06314503
NCT06863194
Data Source & Attribution
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