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NCT Number: NCT01485978

Efficacy and Safety Study 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.

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Key information

Conditions

Renal Insufficiency, Chronic Chronic Disease Collagen Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Disease Attributes Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Kidney Diseases Male Urogenital Diseases Nephritis Nephritis, Hereditary Pathologic Processes Pathological Conditions, Signs and Symptoms Renal Insufficiency Skin and Connective Tissue Diseases Urogenital Abnormalities Urogenital Diseases Urologic Diseases

Age range

24 month–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University Medical Center Goettingen

Göttingen, 37075, Germany

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Definitive diagnosis of Alport syndrome: Kidney biopsy (patient or affected relative/s), and/or mutation analysis (hemizygous X-chromosomal or homozygous autosomal-recessive) and assessment of criteria for clinical diagnosis (haematuria, positive family history regarding kidney diseases, ocular changes, labyrinthine hearing loss)
  • Alport syndrome levels 0, I or II at screening (microhaematuria without microalbuminuria or microalbuminuria [30-300 mg albumin/gCrea]) or proteinuria >300 mg albumin/gCrea with GFR>80ml/min). Patients with Alport stage II are not subject to randomization but are treated opel label.
  • Aged between ≥24 months and <18 years at screening
  • Assent from patient and informed consent from parents/legal guardian

Exclusion criteria

  • Uncertain diagnosis or variants of Alport syndrome such as a heterozygous carrier
  • Alport syndrome levels III, or IV (albuminuria >300 mg/g Crea, creatinine clearance <60 mL/min, or end stage renal failure [ESRF])
  • Known allergies or intolerances to ramipril or related compounds
  • Known contraindication for ACEi-therapy
  • Additional chronic renal, pulmonary or cardiac diseases
  • Pregnancy and lactation

Treatment and study plan

Ramipril

Drug

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.

Placebo to ramipril

Drug

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

Primary outcomes

  1. 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.

  2. 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.

Secondary outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Institut fuer anwendungsorientierte Forschung und klinische Studien GmbH

Other

Collaborators

  • German Federal Ministry of Education and Research
  • University Medical Center Goettingen

Registry information

Official study title

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

Important dates

Study start
2012
Primary completion
2018
Study completion
2019
First posted
Dec 6, 2011
Registry last updated
Jun 17, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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