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

NCT07523581 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: EL-017
• Study Type: interventional
• Target: 24
• Locations: 2 countries
• Sites: 6

Brief 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

Conditions

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

Keywords

Nonsense Mutation; Translational read through

Outcome Measures

Primary Outcomes (2)

• The change in the degree of podocyte foot process effacement

  Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)

  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)

  Baseline to Week 32

Secondary Outcomes (2)

• The percentage change in Urine Protein Creatinine Ratio (UPCR)

  Baseline to Week 16

• The percentage change in Urine Protein Creatinine Ratio (UPCR)

  Baseline to Week 32

Other Outcomes (4)

• The percentage change in estimated Glomerular Filtration Rate (eGFR)

  Baseline to Week 16 and Week 32

• The change in the degree of podocyte foot process effacement

  Baseline to Week 16 and Week 32

• The change in collagen expression

  Baseline to Week 16 and Week 32

Study Arms (2)

Exaluren

EXPERIMENTAL

Drug: Exaluren

Placebo

PLACEBO COMPARATOR

Drug: Exaluren

Interventions

Exaluren DRUG

Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

Exaluren; Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Eloxx Pharmaceuticals, Inc.: lead

Study Sites (6)

Denver Nephrologists PC, Colorado Kidney Care PC

Denver, Colorado, 80220, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, L12 2AP, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Nephritis, Hereditary

Condition Hierarchy (Ancestors)

Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Nephritis; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 5, 2026
• First Posted: April 13, 2026
• Study Start (Estimated): May 30, 2026
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 13, 2026

Record last verified: 2026-04

Locations

US (2); GB (4)