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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06425055
Other study ID # EYP001-208
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date June 6, 2024
Est. completion date October 13, 2025

Study information

Verified date June 2024
Source Enyo Pharma
Contact Isabelle Martin
Phone +33 4 37 70 02 44
Email clinicaltrial@enyopharma.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Recruiting
Enrollment 20
Est. completion date October 13, 2025
Est. primary completion date July 21, 2025
Accepts healthy volunteers No
Gender All
Age group 16 Years to 55 Years
Eligibility 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 of the protocol. Exclusion Criteria: - Is an employee of a site, clinical research organization, vendor, or sponsor involved with this study. - Is pregnant or breastfeeding. - Has participated in any investigational drug study within 60 days prior to D1. - Any clinically significant illness within 30 days before D1 or surgical or medical condition (other than Alport syndrome) that could interfere with the subject's study compliance; confound the study results; impact subject safety. - Any history of active malignancy within the last 1 year before D1. - Any other condition or circumstance that, in the opinion of the investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the subject's safety and well-being. - Has a history of an allergic condition that required the prescription of an emergency epinephrine injection (such as the EpiPen® Auto-Injector). - Any prohibited co-medications within 30 days prior D1. - Has ALT or AST above near normal (>1.5×ULN) at baseline. - Are at high risk for atherosclerotic cardiovascular disease (ASCVD) risk, with an LDL-C level > 160 mg/dL (4.15 mmol/L) and subjects at intermediate risk for ASCVD risk, with a LDL-C level > 190 mg/dL (4.91 mmol/L). - Has moderate or severe hepatic impairment (Child-Pugh score B or C). - Is taking CYP3A4/5 inhibitors or inducers.

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
France Pr Claire Rigothier - CHU De Bordeaux Bordeaux
France Dr Thomas Robert - Hôpital de la Conception Marseille
France Dr Moglie Le Quintrec - Hopital Lapeyronie Montpellier
France Pr. Bertrand Knebelmann - Necker Enfants Malades Paris
Germany Charite Universitatsmedizin Berlin Berlin
Germany University Medicine Goettingen Göttingen
Spain Fundacio Puigvert Barcelona
Spain Hospital Virgen de la Arrixaca El Palmar
Spain Fundacion Jimenez Diaz Madrid
Spain Hospital De Sagunto Sagunto
United States Dr Eric Wallace - University of Alabama Birmingham Alabama
United States Dr Arnold Silva - Boise Kidney & Hypertension Boise Idaho
United States Dr James Simon - Cleveland Clinic Foundation Cleveland Ohio
United States Dr Ankit Mehta - Renal Disease Research Institute Dallas Texas
United States Dr Suneel Udani - NANI Research Hinsdale Illinois
United States Dr Anjay Rastogi - UCLA Health, David Geffen School of Medicine Los Angeles California
United States Dr Andrew Bomback - Columbia University Medical Center New York New York
United States Dr Tingting Li - Washington University Saint Louis Missouri

Sponsors (1)

Lead Sponsor Collaborator
Enyo Pharma

Countries where clinical trial is conducted

United States,  France,  Germany,  Spain, 

Outcome

Type Measure Description Time frame Safety issue
Other Change in albuminuria To determine the effect of vonafexor on and off treatment on exploratory renal biomarkers During on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline
Other Change in proteinuria To determine the effect of vonafexor on and off treatment on exploratory renal biomarkers During on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline
Other Change in Neutrophil Gelatinase Associated Lipocalin (NGAL) To determine the effect of vonafexor on and off treatment on exploratory renal biomarkers During on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline
Primary Number of Treatment-Emergent Adverse Event (TEAE) To assess the safety and tolerability of vonafexor From first dose of treatment until 2 weeks after last dose of treatment, i.e assessed up to 26 weeks
Secondary Change in eGFR To determine the on with the off-treatment effect of three dose levels of vonafexor on renal function During on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline
Secondary Vonafexor plasma concentrations To determine vonafexor plasma concentrations levels During on-treatment period, assessed up to 24 weeks
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