A Study of ELX-02 in Patients With Alport Syndrome

Last updated: December 19, 2022
Sponsor: Eloxx Pharmaceuticals, Inc.
Overall Status: Active - Recruiting

Phase

2

Condition

Kidney Disease

Nephropathy

Alport's Syndrome

Treatment

N/A

Clinical Study ID

NCT05448755
EL-014
  • Ages 6-30
  • All Genders

Study Summary

This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.

In total, up to 8 participants, with a minimum of 3 adults, 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 8 weeks (60 days)

  • a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with adocumented nonsense mutation of Col4A5 in a male or nonsense mutation of Col4A3 orCol4A4 (male or female)
  • The nonsense mutation should be UAG or UGA
  • eGFR>60 ml/min/1.73 m2 (based on CKD-EPI for ages ≥18 and Schwartz formula forparticipants <18)
  • Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500 mg/g]
  • Stable regimen of ACEi/ARB for at least 4 weeks before screening (unless there is acontraindication)

Exclusion

Exclusion Criteria:

  • History of any organ transplantation
  • Mutation consistent with autosomal dominant Alport Syndrome
  • Liver disease characterized by cirrhosis or portal hypertension. Participants withalanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a totalbilirubin 3.0 times the upper limit of normal (ULN) will be excluded
  • History of congestive heart failure diagnosed clinically or with documented leftventricular ejection fraction (LVEF) ≤ 40%
  • History of dialysis

Study Design

Total Participants: 8
Study Start date:
November 28, 2022
Estimated Completion Date:
May 30, 2023

Connect with a study center

  • Monash Medical Center

    Clayton, Victoria 3168
    Australia

    Active - Recruiting

  • Royal Children's Hospital

    Parkville, Victoria 3051
    Australia

    Site Not Available

  • Great Ormond Street Hospital

    London, WC1N 3JH
    United Kingdom

    Active - Recruiting

  • Royal Free Hospital

    London, NW3 2QG
    United Kingdom

    Active - Recruiting

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