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 UCLA
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