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

RecruitingPhase 2Ages 12 Years - anyAllEloxx Pharmaceuticals, Inc.
NCT07523581 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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

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📋 Eligibility rules (as published)
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
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What it tests

  • Exaluren
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Where

11 sites
University of California, Los Angeles
Los Angeles, United States
Stanford University
Stanford, United States
Denver Nephrologists PC, Colorado Kidney Care PC
Denver, United States
University of Minnesota
Minneapolis, United States
Hackensack University Medical Center
Hackensack, United States
University of Pennsylvania
Philadelphia, United States
Renal Associates, P.A.
San Antonio, United States
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom

…and 3 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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