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RECRUITING Phase 2 ClinicalTrials.gov

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

Sponsor: Eloxx Pharmaceuticals, Inc. • ClinicalTrials.gov ID: NCT07523581

Registry updated: 2026-09-30
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Description

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

Interventions

  • Exaluren (Drug)
    Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

Eligibility criteria

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\>40 ml/min/1.73 m2 * Urinary protein based on two spot urine collections \[urine protein/creatinine ratio (UPCR) ≥ 300 mg/g\] * 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

Conditions

Alport Syndrome, X-Linked Alport Syndrome, Autosomal Recessive

Locations

  • University of California, Los Angeles — Los Angeles, California, United States
  • Stanford University — Stanford, California, United States
  • Denver Nephrologists PC, Colorado Kidney Care PC — Denver, Colorado, United States
  • University of Minnesota — Minneapolis, Minnesota, United States
  • Hackensack University Medical Center — Hackensack, New Jersey, United States
  • University of Pennsylvania — Philadelphia, Pennsylvania, United States
  • Renal Associates, P.A. — San Antonio, Texas, United States
  • Alder Hey Children's NHS Foundation Trust — Liverpool, United Kingdom
  • Royal Free Hospital — London, United Kingdom
  • Great Ormond Street Hospital — London, United Kingdom
  • Royal Manchester Children's Hospital — Manchester, United Kingdom
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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record