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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Early Prospective Therapy Trial to Delay Renal Failure in Children With Alport Syndrome

Brief 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.


Detailed Summary:
Sponsor: Institut fuer anwendungsorientierte Forschung und klinische Studien GmbH

Current Primary Outcome:

  • Time to next disease level [ Time Frame: within 3 years ]
    Time to progression of Alport Syndrome to the next disease level within 3 years under ramipril treatment compared to placebo, for all randomised patients.
  • Incidence of Adverse Drug Events before progression [ Time Frame: within 3 years ]
    Incidence of adverse drug events (ADEs, e.g., angioedema, acute renal failure, hyperkalaemia) under ramipril treatment before disease progression compared to placebo before disease progression, for all randomised patients.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Albuminuria after three years [ Time Frame: after 3 years ]
    Albuminuria after 3 years corrected for baseline albuminuria for patients randomised to receive ramipril compared to placebo.
  • Adverse Drug Events over three years [ Time Frame: after 3 years ]
    Incidence of ADEs (e.g., angioedema, acute renal failure, hyperkalaemia) during 3 years of treatment for patients randomised to receive ramipril compared to placebo.


Original Secondary Outcome: Same as current

Information By: Institut fuer anwendungsorientierte Forschung und klinische Studien GmbH

Dates:
Date Received: December 2, 2011
Date Started: March 2012
Date Completion: August 2019
Last Updated: August 7, 2014
Last Verified: August 2014