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

NCT07523581 — Phase 2, Recruiting, 24 participants, Eloxx Pharmaceuticals, Inc.

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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

Registry	NCT07523581
Status	Recruiting
Phase	Phase 2
Study type	Interventional; randomized, sequential, masking: quadruple, treatment
Enrollment	24 (Estimated)
Sponsor	Eloxx Pharmaceuticals, Inc. (Industry)
Conditions	Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
Interventions	Exaluren (Drug)
Dates	start 2026-06-30, primary completion 2027-06-30 (estimated), completion 2027-12-31 (estimated), first posted 2026-04-13, last updated 2026-09-30
Locations	11 sites in United States, United Kingdom

-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

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+Arms and interventions

Arm	Type	Interventions	Description
Exaluren	Experimental	Drug: Exaluren
Placebo	Placebo comparator	Drug: Exaluren

Exaluren (Drug): Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

+Outcome measures

Primary outcomes

The change in the degree of podocyte foot process effacement [Baseline to Week 16]: Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
The change in the degree of podocyte foot process effacement [Baseline to Week 32]: Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)

Secondary outcomes

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

Other outcomes

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

+Eligibility

Sex: All, ages: 12 Years to any

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>40 ml/min/1.73 m2
* Urinary protein based on two spot urine collections \[urine protein/creatinine ratio (UPCR) ≥ 300 mg/g\]
* 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

+Locations (11)

Facility	City	Country	Status
University of California, Los Angeles	Los Angeles, California	United States	Not yet recruiting
Stanford University	Stanford, California	United States	Not yet recruiting
Denver Nephrologists PC, Colorado Kidney Care PC	Denver, Colorado	United States	Recruiting
University of Minnesota	Minneapolis, Minnesota	United States	Not yet recruiting
Hackensack University Medical Center	Hackensack, New Jersey	United States	Not yet recruiting
University of Pennsylvania	Philadelphia, Pennsylvania	United States	Not yet recruiting
Renal Associates, P.A.	San Antonio, Texas	United States	Not yet recruiting
Alder Hey Children's NHS Foundation Trust	Liverpool	United Kingdom	Not yet recruiting
Royal Free Hospital	London	United Kingdom	Recruiting
Great Ormond Street Hospital	London	United Kingdom	Recruiting
Royal Manchester Children's Hospital	Manchester	United Kingdom	Not yet recruiting