NCT07523581

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

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

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Eloxx Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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. 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

Conditions

• Alport Syndrome, X-Linked
• Alport Syndrome, Autosomal Recessive

Interventions

Type	Name	Description
DRUG	Exaluren	Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

Timeline

• Start date: 2026-05-30
• Primary completion: 2027-06-30
• Completion: 2027-12-31
• First posted: 2026-04-13
• Last updated: 2026-04-13

Locations

6 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study