This study is not yet accepting patients

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

a study on Alport Syndrome

Summary

Eligibility
for people ages 12 years and up (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
study ends around

Description

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

Official Title

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

Keywords

Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive, Nonsense Mutation, Translational read through, Hereditary Nephritis, Exaluren

Eligibility

You can join if…

Open to people ages 12 years and up

  • 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>45 ml/min/1.73 m2
  • Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500 mg/g]
  • Stable regimen of ACEi/ARB for at least 12 weeks before Day 1

You CAN'T join if...

  • 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

Locations

  • University of California, Los Angeles
    Los Angeles California 90095 United States
  • Stanford University
    Stanford California 94305 United States

Details

Status
not yet accepting patients
Start Date
Completion Date
(estimated)
Sponsor
Eloxx Pharmaceuticals, Inc.
ID
NCT07523581
Phase
Phase 2 Alport Syndrome Research Study
Study Type
Interventional
Participants
Expecting 24 study participants
Last Updated