Early prospective therapy trial to delay renal failure in children with Alport syndrome. - EARLY PRO-TECT Alport

Phase 1

• Conditions: Alport's syndrome; MedDRA version: 19.1Level: PTClassification code 10001843Term: Alport's syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2010-024300-10-DE
• Lead Sponsor: niversity Medical Center Göttingen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2011-12-12
• Study Completion: 2019-03-25
• Last Update Posted: 8 March 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 66

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

Are the trial subjects under 18? yes
Number of subjects for this age range: 120
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Uncertain diagnosis or variants of Alport syndrome such as a heterozygous carrier

- Alport syndrome levels III, or IV (creatinine clearance <80 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To investigate if the treatment of Alport's syndrome with the ACE inhibitor ramipril from early stages of disease is safe and significantly slows disease progression to renal failure.;Secondary Objective: ;Primary end point(s): Primary Efficacy Endpoint: Time to progression of Alport Syndrome to the next disease level under ramipril treatment compared to placebo, for all randomised patients.<br><br>Primary Safety Endpoint: 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.<br>;Timepoint(s) of evaluation of this end point: Primary Efficacy Endpoint: within up to 6 years, until disease progression<br><br>Primary Safety Endpoint: within up to 6 years, until disease progression

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary Efficacy Endpoint: Albuminuria after end of treatment corrected for baseline albuminuria for patients randomised to receive ramipril compared to placebo.<br><br>Secondary Safety Endpoint: Incidence of ADEs (e.g., angioedema, acute renal failure, hyperkalaemia) during treatment for patients randomised to receive ramipril compared to placebo.<br>;Timepoint(s) of evaluation of this end point: Secondary Efficacy Endpoint: after up to 6 years<br><br>Secondary Safety Endpoint: after up to 6 years