A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes

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.

CT.gov Identifier
NCT07523581
EudraCT Identifier
N/A
CTIS:
N/A
Sponsor
Eloxx Pharmaceuticals
Collaborator
N/A
Study Contact Information
N/A
Recruiting
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Study Details

Diseases
Alport syndrome
Study Drug
Exaluren sulfate
Genes
COL4A4
COL4A3
COL4A5
Study Dates
Jul 2026 - Jun 2027
Sex
Female & Male
Age
12+ years
Inclusion and Exclusion 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>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

Protocol 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.

Study Locations

To view all trial locations, click here

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