Efficacy and Safety Study to Delay Renal Failure in Children With Alport Syndrome

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.

Conditions

• Renal Insufficiency, Chronic

Interventions

DRUG

Ramipril

Ramipril (Delix) tablets containing 2.5 mg ramipril, oral application with 1 to 6 mg per body surface area ramipril once daily for 3 years.

DRUG

placebo to ramipril

Oral application of placebo to ramipril, once daily with 1 to 6 mg per body surface area for 3 years or until disease progression.

DRUG

Ramipril

Oral treatment with 1 to 6 mg per body surface area ramipril once daily for 3 years as per protocol.

Sponsors & Collaborators

• University Medical Center Goettingen

  collaborator OTHER

• German Federal Ministry of Education and Research

  collaborator OTHER_GOV

• Institut fuer anwendungsorientierte Forschung und klinische Studien GmbH

  lead OTHER

Principal Investigators

• Oliver Gross, Prof. · University Medical Center Goettingen, Department Nephrology and Rheumatology

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Model

PARALLEL

Eligibility

Min Age

24 Months

Max Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2012-03-31

Primary Completion

2018-09-30

Completion

2019-03-31

Countries

• Germany

Study Locations