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

April 5, 2026 updated by: Eloxx Pharmaceuticals, Inc.

A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes

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 Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

24

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • Liverpool, United Kingdom, L12 2AP
      • London, United Kingdom, Nw3 2QG
        • Royal Free Hospital
        • Contact:
      • London, United Kingdom, WC1N 3JH
      • Manchester, United Kingdom, M13 9WL
        • Royal Manchester Children's Hospital
        • Contact:
  • United States
    • Colorado
      • Denver, Colorado, United States, 80220
        • Denver Nephrologists PC, Colorado Kidney Care PC
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Exaluren
Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)
Experimental: Exaluren
Drug: Exaluren
Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The change in the degree of podocyte foot process effacement
Time Frame: Baseline to Week 16
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
Time Frame: Baseline to Week 32
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
Baseline to Week 32

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Time Frame: Baseline to Week 16
Calculated from geometric mean of 3 consecutive days of first void urine collection
Baseline to Week 16
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Time Frame: Baseline to Week 32
Calculated from geometric mean of 3 consecutive days of first void urine collection
Baseline to Week 32

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
The percentage change in estimated Glomerular Filtration Rate (eGFR)
Time Frame: Baseline to Week 16 and Week 32
Change in eGFR level
Baseline to Week 16 and Week 32
The change in the degree of podocyte foot process effacement
Time Frame: Baseline to Week 16 and Week 32
Measured by the change in Foot Process Width (FPW)
Baseline to Week 16 and Week 32
The change in collagen expression
Time Frame: Baseline to Week 16 and Week 32
Measured by structural changes
Baseline to Week 16 and Week 32
Evaluate the safety and tolerability of Exaluren
Time Frame: Screening to End of Study (Week 36)
The incidence and characteristics of adverse events (AEs)
Screening to End of Study (Week 36)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Eloxx Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

May 30, 2026

Primary Completion (Estimated)

June 30, 2027

Study Completion (Estimated)

December 31, 2027

Study Registration Dates

First Submitted

April 5, 2026

First Submitted That Met QC Criteria

April 5, 2026

First Posted (Actual)

April 13, 2026

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 5, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EL-017

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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