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NCT Number: NCT07523581

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

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

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Key information

Conditions

Alport Syndrome, X-Linked Alport Syndrome, Autosomal Recessive Collagen Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Kidney Diseases Male Urogenital Diseases Nephritis Nephritis, Hereditary Skin and Connective Tissue Diseases Urogenital Abnormalities Urogenital Diseases Urologic Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Exaluren

Drug

Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

Primary outcomes

  1. 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)

  2. 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)

Secondary outcomes

  1. 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

  2. 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

Other outcomes

  1. The percentage change in estimated Glomerular Filtration Rate (eGFR)

    Time frame: Baseline to Week 16 and Week 32

    Change in eGFR level

  2. 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)

  3. The change in collagen expression

    Time frame: Baseline to Week 16 and Week 32

    Measured by structural changes

  4. Evaluate the safety and tolerability of Exaluren

    Time frame: Screening to End of Study (Week 36)

    The incidence and characteristics of adverse events (AEs)

Sponsors and collaborators

Lead sponsor

Eloxx Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Apr 13, 2026
Registry last updated
Jul 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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