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NCT07523581PHASE2Recruiting

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

Eloxx Pharmaceuticals, Inc.

Start Date

6/30/2026

Completion Date

12/31/2027

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

Eligibility Criteria

Age Range: 12 years to No maximum

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

Interventions

DRUG

Exaluren

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Conditions

Alport Syndrome, X-LinkedAlport Syndrome, Autosomal Recessive

Locations

University of California, Los Angeles

Los Angeles, California 90095

United States

Stanford University

Stanford, California 94305

United States

Denver Nephrologists PC, Colorado Kidney Care PC

Denver, Colorado 80220

United States

University of Minnesota

Minneapolis, Minnesota 55455

United States

Hackensack University Medical Center

Hackensack, New Jersey 07601

United States

Cleveland Clinic

Cleveland, Ohio 44195

United States

University of Pennsylvania

Philadelphia, Pennsylvania 19104

United States

Renal Associates, P.A.

San Antonio, Texas 78109

United States

Alder Hey Children's NHS Foundation Trust

Liverpool, L12 2AP

United Kingdom

Royal Free Hospital

London, NW3 2QG

United Kingdom

Great Ormond Street Hospital

London, WC1N 3JH

United Kingdom

Royal Manchester Children's Hospital

Manchester, M13 9WL

United Kingdom