Informations générales (source: ClinicalTrials.gov)
Vonafexor Fixed Dose-escalation Safety and Proof-of-concept Study in Patients With at Risk of Progression Alport Syndrome (ALPESTRIA-1)
Interventional
Phase 2
Enyo Pharma (Voir sur ClinicalTrials)
août 2024
octobre 2025
04 avril 2025
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.
Etablissements
| Les établissements d'Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| AP-HP - Hôpital Necker-Enfants Malades | Contact (sur clinicalTrials) | ||||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| Dr Moglie Le Quintrec - Hopital Lapeyronie - 34090 - Montpellier - France | Contact (sur clinicalTrials) | ||||
| Pr Claire Rigothier - CHU De Bordeaux - 33076 - Bordeaux - France | Contact (sur clinicalTrials) | ||||
Critères
Tous
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.
- 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.
- 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.