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Purpose

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

Conditions

Eligibility

Eligible Ages
Over 12 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Exaluren
  • Drug: Exaluren
    Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)
Placebo Comparator
Placebo
  • Drug: Exaluren
    Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

Recruiting Locations

Denver Nephrologists PC, Colorado Kidney Care PC
Denver, Colorado 80220
Contact:
Laura Kooienga
lkooienga@cokidneycare.com

More Details

Status
Recruiting
Sponsor
Eloxx Pharmaceuticals, Inc.

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.