Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1)
- Conditions
- Alport Syndrome
- Registration Number
- NCT06425055
- Lead Sponsor
- Enyo Pharma
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria:<br><br> - Signed informed consent (also for legal representatives, as applicable in the US for<br> under eighteen patients).<br><br> - Has confirmed diagnosis of Alport syndrome: clinical diagnosis (haematuria, family<br> history, hearing loss, ocular change) OR a kidney biopsy showing glomerular basement<br> membrane abnormalities consistent with AS, AND Genetic confirmation of AS.<br><br> - Has eGFR between = 30 and < 90 ml/min/1.73m2.<br><br> - Has increased albuminuria criteria i.e. UACR = 300 mg/g.<br><br> - If on an angiotensin converting enzyme inhibitor (ACEi) and/or angiotensin receptor<br> blocker (ARB), should be on a stable well tolerated treatment during at least the 60<br> days prior D1.<br><br> - If on Sodium-Glucose Transport Protein 2 (SGLT2), should be on stable well tolerated<br> treatment with SGLT2 during at least 60 days prior D1.<br><br> - If patient has a history of arterial hypertension, should be on stable<br> anti-hypertensive therapy for at least 60 days prior to D1 and deemed controlled by<br> the investigator at screening and D1.<br><br> - Sexually active female subjects of childbearing potential and sexually mature male<br> subjects must use two acceptable effective methods of contraception for the entire<br> duration of the study and for at least 6 weeks after last dose.<br><br> - Has negative results for hepatitis B surface antigen (HBsAg), hepatitis C virus<br> (HCV) antibody, and human immunodeficiency virus (HIV).<br><br> - Is able to understand all study procedures in the informed consent form (ICF) and<br> willing to comply with all aspects of the protocol.<br><br>Exclusion Criteria:<br><br> - Is an employee of a site, clinical research organization, vendor, or sponsor<br> involved with this study.<br><br> - Is pregnant or breastfeeding.<br><br> - Has participated in any investigational drug study within 60 days prior to D1.<br><br> - Any clinically significant illness within 30 days before D1 or surgical or medical<br> condition (other than Alport syndrome) that could interfere with the subject's study<br> compliance; confound the study results; impact subject safety.<br><br> - Any history of active malignancy within the last 1 year before D1.<br><br> - Any other condition or circumstance that, in the opinion of the investigator, may<br> make the subject unlikely to complete the study or comply with study procedures and<br> requirements, or may pose a risk to the subject's safety and well-being.<br><br> - Has a history of an allergic condition that required the prescription of an<br> emergency epinephrine injection (such as the EpiPen® Auto-Injector).<br><br> - Any prohibited co-medications within 30 days prior D1.<br><br> - Has ALT or AST above near normal (>1.5×ULN) at baseline.<br><br> - Are at high risk for atherosclerotic cardiovascular disease (ASCVD) risk, with an<br> LDL-C level > 160 mg/dL (4.15 mmol/L) and subjects at intermediate risk for ASCVD<br> risk, with a LDL-C level > 190 mg/dL (4.91 mmol/L).<br><br> - Has moderate or severe hepatic impairment (Child-Pugh score B or C).<br><br> - Is taking CYP3A4/5 inhibitors or inducers.
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Treatment-Emergent Adverse Event (TEAE)
- Secondary Outcome Measures
Name Time Method Change in eGFR;Vonafexor plasma concentrations