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)
University of California, Los Angeles
Los Angeles, California, United States
NOT_YET_RECRUITINGStanford University
Stanford, California, United States
NOT_YET_RECRUITINGDenver Nephrologists PC, Colorado Kidney Care PC
Denver, Colorado, United States
RECRUITINGUniversity of Minnesota
Minneapolis, Minnesota, United States
NOT_YET_RECRUITINGHackensack University Medical Center
Hackensack, New Jersey, United States
NOT_YET_RECRUITINGCleveland Clinic
Cleveland, Ohio, United States
NOT_YET_RECRUITINGUniversity of Pennsylvania
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGRenal Associates, P.A.
San Antonio, Texas, United States
NOT_YET_RECRUITINGAlder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom
NOT_YET_RECRUITINGRoyal Free Hospital
London, United Kingdom
RECRUITING...and 2 more locations
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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