Recruiting
Phase 2

Exaluren

Sponsor:

Eloxx Pharmaceuticals, Inc.

Code:

NCT07523581

Conditions

Alport Syndrome, X-Linked

Alport Syndrome, Autosomal Recessive

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Interventions

Exaluren

Study Details

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, X-Linked

Alport Syndrome, Autosomal Recessive

Study ID

NCT07523581

Start date

Jun 30, 2026

Status verified date

Jul, 2026

Completion date

Dec 31, 2027

Anticipated

Primary completion date

Jun 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

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

Enrollment

24 participants

Anticipated

Allocation

Randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Exaluren

placebo comparator: Placebo

Interventions

Exaluren

Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

Primary outcome measure

  • The change in the degree of podocyte foot process effacement [ Time Frame: Baseline to Week 16 ]
  • The change in the degree of podocyte foot process effacement [ Time Frame: Baseline to Week 32 ]

Central Contacts and Locations

Locations

Denver Nephrologists PC, Colorado Kidney Care PC

Recruiting

Denver, Colorado, United States, 80220

Contacts

Laura Kooienga

303-364-4775lkooienga@cokidneycare.com

More Information

Sponsor

Eloxx Pharmaceuticals, Inc.

Last update posted

Jul 15, 2026

Last verified

Jul, 2026

Keywords

  • Nonsense Mutation
  • Translational read through

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-27. This information was provided to ClinicalTrials.gov by Eloxx Pharmaceuticals, Inc. on 2026-07-15. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.