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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
26
* 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
Dr Eric Wallace - University of Alabama
Birmingham, Alabama, United States
Dr Anjay Rastogi - UCLA Health, David Geffen School of Medicine
Los Angeles, California, United States
Dr Arnold Silva - Boise Kidney & Hypertension
Boise, Idaho, United States
Number of Treatment-Emergent Adverse Event (TEAE)
To assess the safety and tolerability of vonafexor
Time frame: From first dose of treatment until 2 weeks after last dose of treatment, i.e assessed up to 26 weeks
Change in eGFR
To determine the on with the off-treatment effect of three dose levels of vonafexor on renal function
Time frame: During on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline
Vonafexor plasma concentrations
To determine vonafexor plasma concentrations levels
Time frame: During on-treatment period, assessed up to 24 weeks
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Dr Suneel Udani - NANI Research
Hinsdale, Illinois, United States
Dr Tingting Li - Washington University
St Louis, Missouri, United States
Dr James Simon - Cleveland Clinic Foundation
Cleveland, Ohio, United States
Dr Ankit Mehta - Renal Disease Research Institute
Dallas, Texas, United States
Pr Claire Rigothier - CHU De Bordeaux
Bordeaux, France
Dr Moglie Le Quintrec - Hopital Lapeyronie
Montpellier, France
Pr. Bertrand Knebelmann - Necker Enfants Malades
Paris, France
...and 3 more locations