EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

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

  • Alport Syndrome, X-Linked
  • Alport Syndrome, Autosomal Recessive

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