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

Recruiting now · Phase 2 · Has a placebo group

Conditions studied: Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive

In brief

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

Key facts

Study ID

NCT07523581

Run by

Eloxx Pharmaceuticals, Inc.

People needed

24

Starts

2026-06-30

Expected to finish

2027-12-31

Last updated by the study team

2026-07-15

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Royal Free Hospital — London, United Kingdom (enrolling)
• Great Ormond Street Hospital — London, United Kingdom (enrolling)
• Denver Nephrologists PC, Colorado Kidney Care PC — Denver, Colorado, United States (enrolling)
• Hackensack University Medical Center — Hackensack, New Jersey, United States
• Cleveland Clinic — Cleveland, Ohio, 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
• University of California, Los Angeles — Los Angeles, California, United States
• Royal Manchester Children's Hospital — Manchester, United Kingdom
• Stanford University — Stanford, California, United States
• University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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