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
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
24
Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)
Denver Nephrologists PC, Colorado Kidney Care PC
Denver, Colorado, United States
University of Minnesota
Minneapolis, Minnesota, United States
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom
Royal Free Hospital
London, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom
The change in the degree of podocyte foot process effacement
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
Time frame: Baseline to Week 16
The change in the degree of podocyte foot process effacement
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
Time frame: Baseline to Week 32
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Calculated from geometric mean of 3 consecutive days of first void urine collection
Time frame: Baseline to Week 16
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Calculated from geometric mean of 3 consecutive days of first void urine collection
Time frame: Baseline to Week 32
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