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.
Who can participate
Age range16 Years – 55 Years
SexALL
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Inclusion Criteria:
* Signed informed consent (also for legal representatives, as applicable in the US for under eighteen patients).
* Has confirmed diagnosis of Alport syndrome: clinical diagnosis (haematuria, family history, hearing loss, ocular change) OR a kidney biopsy showing glomerular basement membrane abnormalities consistent with AS, AND Genetic confirmation of AS.
* Has eGFR between ≥ 30 and \< 90 ml/min/1.73m2.
* Has increased albuminuria criteria i.e. UACR ≥ 300 mg/g.
* If on an angiotensin converting enzyme inhibitor (ACEi) and/or angiotensin receptor blocker (ARB), should be on a stable well tolerated treatment during at least the 60 days prior D1.
* If on Sodium-Glucose Transport Protein 2 (SGLT2), should be on stable well tolerated treatment with SGLT2 during at least 60 days prior D1.
* If patient has a history of arterial hypertension, should be on stable anti-hypertensive therapy for at least 60 days prior to D1 and deemed controlled by the investigator at screening and D1.
* Sexually active female subjects of childbearing potential and sexually mature male subjects must use two acceptable effective methods of contraception for the entire duration of the study and for at least 6 weeks after last dose.
* Has negative results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, and human immunodeficiency virus (HIV).
* Is able to understand all study procedures in the informed consent form (ICF) and willing to comply with all aspects o…
What they're measuring
1
Number of Treatment-Emergent Adverse Event (TEAE)
Timeframe: From first dose of treatment until 2 weeks after last dose of treatment, i.e assessed up to 26 weeks