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

NCT07523581 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2026-07-15

No results posted yet for this study

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

DRUG

Exaluren

Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

Sponsors & Collaborators

  • Eloxx Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-30
Primary Completion
2027-06-30
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07523581 on ClinicalTrials.gov