Not Yet RecruitingPhase 2

EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

United States, United Kingdom24 participantsStarted 2026-05-30

Plain-language summary

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 weeks. Part 2: all patients across both randomized arms receive exaluren for 16 additional weeks. * a safety/efficacy Follow-up Period of 4 weeks after the last treatment

Who can participate

Age range12 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 Exclusion Criteria: * History of any organ transplantation * Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 1.5 times the upper limit of normal (ULN) will be excluded * History of dialysis * Acute kidney injury within 4 weeks before screening * Active dizziness

What they're measuring

The change in the degree of podocyte foot process effacement

Timeframe: Baseline to Week 16

The change in the degree of podocyte foot process effacement

Timeframe: Baseline to Week 32

Trial details

NCT IDNCT07523581

SponsorEloxx Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-06-30