NCT05448755

A Study of ELX-02 in Patients With Alport Syndrome

A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients With Alport Syndrome With Col4A5 and Col4A3/4 Nonsense Mutation

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Eloxx Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 6 Years – 30 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Alport Syndrome

Interventions

Type	Name	Description
DRUG	ELX-02	ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaryotic ribosomal selective glycoside (ERSG).

Timeline

• Start date: 2022-11-28
• Primary completion: 2023-09-11
• Completion: 2023-09-11
• First posted: 2022-07-07
• Last updated: 2026-02-24
• Results posted: 2026-02-24

Locations

4 sites across 2 countries: Australia, United Kingdom