NCT06425055

Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1)

Vonafexor Fixed Dose-escalation Safety and Proof-of-concept Study in Patients With at Risk of Progression Alport Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: Enyo Pharma · Industry
• Sex: All
• Age: 16 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

This study is a proof-of-concept trial of vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome.

Detailed description

This is a multicenter study and several clinical sites and countries will be involved. This single arm, fixed dose escalation, open-label, non-randomized study will evaluate three dose levels of vonafexor on safety, tolerability and their effect on kidney function and renal biomarkers in 20 patients with AS at risk of progression. The total duration of study for a participant will be up to 40 weeks and include a screening period, a treatment period of 24 weeks and a follow-up period of 12 weeks.

Conditions

• Alport Syndrome

Interventions

Type	Name	Description
DRUG	Vonafexor	* One tablet of a low dose of vonafexor QD from Day 1 to Week 4 * One tablet of a medium dose of vonafexor QD from Week 5 to Week 8 * One tablet of a high dose of vonafexor QD from Week 9 to Week 24

Timeline

• Start date: 2024-08-01
• Primary completion: 2025-09-04
• Completion: 2025-11-20
• First posted: 2024-05-22
• Last updated: 2026-02-09

Locations

13 sites across 3 countries: United States, France, Spain

Regulatory

• FDA-regulated drug study