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Vonafexor fixed dose-escalation safety and proof-of-concept study in patients with at risk of progression Alport syndrome
EU CTIS ID: 2023-509638-20-00
What this study is testing
To assess the safety and tolerability of vonafexor (on and off treatment) in at risk of progression Alport syndrome patients.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Subject signed informed consent and legal representatives signed informed consent as applicable for United States (US) under eighteen patients.
- Sexually active female subjects of childbearing potential and sexually mature male subjects must agree to 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)
- Male and female patients ≥ 18 years (in US accepted lower limit age is ≥ 16 years) and ≤ 55 years.
- Has confirmed diagnosis of Alport syndrome: A. Clinical diagnosis (haematuria, family history, hearing loss, ocular change), OR a kidney biopsy showing glomerular basement membrane abnormalities (e.g., significant thinning, thickening, irregularity or lucencies) consistent with AS, AND B. Genetic confirmation of AS (medical history or genotyped during screening)
- Has eGFR between ≥ 30 and < 90 ml/min/1.73m2.
You likely can't join if
- Is pregnant or breastfeeding.
- Has moderate or severe hepatic impairment (Child-Pugh score B or C).
- Is taking CYP3A4/5 inhibitors or inducers.
- 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 (history of localized basal cell or squamous cell carcinoma and cervical carcinoma in situ that has been excised/appropriately treated or a fully excised malignant lesion with a low probability of recurrence will not be considered exclusionary).
See the full eligibility criteria
Who can join
- Subject signed informed consent and legal representatives signed informed consent as applicable for United States (US) under eighteen patients.
- Sexually active female subjects of childbearing potential and sexually mature male subjects must agree to 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)
- Male and female patients ≥ 18 years (in US accepted lower limit age is ≥ 16 years) and ≤ 55 years.
- Has confirmed diagnosis of Alport syndrome: A. Clinical diagnosis (haematuria, family history, hearing loss, ocular change), OR a kidney biopsy showing glomerular basement membrane abnormalities (e.g., significant thinning, thickening, irregularity or lucencies) consistent with AS, AND B. Genetic confirmation of AS (medical history or genotyped during screening)
- 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
What rules you out
- Is pregnant or breastfeeding.
- Has moderate or severe hepatic impairment (Child-Pugh score B or C).
- Is taking CYP3A4/5 inhibitors or inducers.
- 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 (history of localized basal cell or squamous cell carcinoma and cervical carcinoma in situ that has been excised/appropriately treated or a fully excised malignant lesion with a low probability of recurrence will not be considered exclusionary).
- 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 as per section 5.2.3 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) (Lloyd-Jones DM et al., 2019)23.
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.