EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
Start Date
6/30/2026
Completion Date
12/31/2027
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 Criteria
Age Range: 12 years to No maximum
Interventions
Exaluren
Conditions
Locations
University of California, Los Angeles
Los Angeles, California 90095
United States
Stanford University
Stanford, California 94305
United States
Denver Nephrologists PC, Colorado Kidney Care PC
Denver, Colorado 80220
United States
University of Minnesota
Minneapolis, Minnesota 55455
United States
Hackensack University Medical Center
Hackensack, New Jersey 07601
United States
Cleveland Clinic
Cleveland, Ohio 44195
United States
University of Pennsylvania
Philadelphia, Pennsylvania 19104
United States
Renal Associates, P.A.
San Antonio, Texas 78109
United States
Alder Hey Children's NHS Foundation Trust
Liverpool, L12 2AP
United Kingdom
Royal Free Hospital
London, NW3 2QG
United Kingdom
Great Ormond Street Hospital
London, WC1N 3JH
United Kingdom
Royal Manchester Children's Hospital
Manchester, M13 9WL
United Kingdom