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

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

NCT Number	NCT07523581
Lead Sponsor	Eloxx Pharmaceuticals, Inc.
Conditions	Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
Enrollment	24 participants
Start Date	2026-06-30
Primary Completion	2027-06-30 (estimated)
Study Completion	2027-12-31 (estimated)
Updated on ClinicalTrials.gov	2026-07-15