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

RecruitingPhase 2

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

Who can join

Ages 12 and older · All sexes

Full 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

About the study

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

What is being tested

Exaluren (drug)

Sponsor: Eloxx Pharmaceuticals, Inc. · Participants: 24 · Started: Jun 30, 2026

Contact the study team

Official record on ClinicalTrials.gov — NCT07523581

Locations in the U.S.

California	University of California, Los Angeles, Los Angeles (Not yet recruiting) Stanford University, Stanford (Not yet recruiting)
Colorado	Denver Nephrologists PC, Colorado Kidney Care PC, Denver
Minnesota	University of Minnesota, Minneapolis (Not yet recruiting)
New Jersey	Hackensack University Medical Center, Hackensack (Not yet recruiting)
Pennsylvania	University of Pennsylvania, Philadelphia (Not yet recruiting)
Texas	Renal Associates, P.A., San Antonio (Not yet recruiting)

From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.