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

Phase 3

Recruiting

Conditions: MedDRA - 10001843 (Alport`s syndrome)
Q87.8
Other specified congenital malformation syndromes, not elsewhere classified

Registration Number: DRKS00003624

Lead Sponsor: niversitätsmedizin Göttingen

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 120

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 or I at screening (microhaematuria without microalbuminuria or microalbuminuria [30-300 mg albumin/gCrea]).
- 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 II, , 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

Study & Design

Study Type: interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary Efficacy Endpoint: Time to progression of Alport Syndrome to the next disease level within 3 years 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>

Secondary Outcome Measures

Name	Time	Method
Secondary Efficacy Endpoint: Albuminuria after 3 years 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 3 years of treatment for patients randomised to receive ramipril compared to placebo.<br>