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

Condition(s) studied

Investigational drug(s) / intervention(s)

Exaluren: Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

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

Eligibility

Primary outcome measure(s)

• The change in the degree of podocyte foot process effacement — Baseline to Week 16
  Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
• The change in the degree of podocyte foot process effacement — Baseline to Week 32
  Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)

Trial sites (12)