ClinicaliQ Trial Snapshot
- EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety — Recruiting • Phase II • NCT07523581.
- What is being tested: Exaluren, a therapeutic agent designed to enable read-through of nonsense mutations in COL4A3/4/5 genes, is being evaluated in a randomized, double-blind, placebo-controlled trial for safety and efficacy in Alport Syndrome patients with this specific genetic mutation.
- Patient eligibility overview: The trial targets approximately 24 patients aged 12 years and older with genetically confirmed Alport Syndrome carrying nonsense mutations in COL4A3/4/5 genes, representing a defined subset of the Alport Syndrome population.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
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What This Trial Is Studying
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes. Targeted 24 patients aged 12 and older will be enrolled in the trial. The study will be comprised of the following periods for each participant: * a Screening period of up to 6 weeks (42 days) * a total Treatment Period of exaluren 0.75 mg/kg or placebo administered daily subcutaneously for 32 weeks: Part 1: patients are randomized to either exaluren or placebo for 16…
Eligibility Snapshot
- : * A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with a documented nonsense mutation of COL4A5 in a male or nonsense mutation of COL4A3 or COL4A4 (male or female) * eGFR>45 ml/min/1.73 m2 * Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500 mg/g] * Stable regimen of ACEi/ARB for at least 12 weeks before Day 1
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.