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Study on the Safety of Vonafexor for Patients with Alport Syndrome at Risk of Progression

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What is this study about?

This clinical trial is focused on studying Alport syndrome, a genetic condition that can lead to kidney disease, hearing loss, and eye abnormalities. The trial will test a medication called Vonafexor, which is taken as a tablet. The purpose of the study is to assess the safety and tolerability of Vonafexor in patients who are at risk of progression of Alport syndrome.

Participants in the study will receive Vonafexor in a fixed dose-escalation manner, meaning the dose may be gradually increased to find the most suitable level. Some participants may receive a placebo instead of the actual medication. The study will monitor the participants’ health through regular check-ups, including physical examinations and laboratory tests, to observe any changes in their condition while on the treatment and after the treatment period ends.

The trial will also measure the levels of Vonafexor in the blood to ensure they match expected concentrations. This will help researchers understand how the medication is processed in the body. The study aims to provide valuable information on the potential benefits and any side effects of Vonafexor for people with Alport syndrome.

1 joining the study

Upon joining the study, the patient must have signed an informed consent form. This is a document that explains the study and confirms the patient’s willingness to participate.

The patient must meet specific criteria, such as being between 18 and 55 years old, having a confirmed diagnosis of Alport syndrome, and having certain kidney function levels.

2 initial assessment

The patient undergoes an initial assessment to confirm eligibility. This includes tests for hepatitis B, hepatitis C, and HIV, as well as a review of medical history and current medications.

3 treatment phase

The patient begins taking vonafexor, which is administered orally in tablet form. The dosage and frequency are determined by the study protocol.

The treatment phase involves regular monitoring of the patient’s health, including physical examinations, vital signs, and laboratory tests.

4 monitoring and evaluation

Throughout the treatment phase, the patient is monitored for any adverse effects. This includes tracking any new symptoms or changes in health status.

The patient’s kidney function and vonafexor plasma levels are evaluated at specific intervals to assess the drug’s impact.

5 end of treatment

After completing the treatment phase, the patient stops taking vonafexor and continues to be monitored for a period to observe any changes in health status.

The study aims to assess the safety and tolerability of vonafexor both during and after treatment.

6 final assessment

A final assessment is conducted to evaluate the overall effects of the treatment. This includes a comparison of health indicators from before and after the study.

Who Can Join the Study?

  • The patient must have signed an informed consent form. If the patient is under eighteen in the United States, their legal representatives must also sign.
  • Sexually active females who can have children and sexually mature males must agree to use two effective methods of birth control during the study and for at least 6 weeks after the last dose.
  • The patient must test negative for hepatitis B, hepatitis C, and HIV.
  • The patient must be between 18 and 55 years old. In the United States, patients as young as 16 can participate.
  • The patient must have a confirmed diagnosis of Alport Syndrome. This can be through clinical signs like blood in urine, family history, hearing loss, or eye changes, or through a kidney biopsy showing specific changes. Genetic confirmation is also required.
  • The patient must have an eGFR (a measure of kidney function) between 30 and less than 90 ml/min/1.73m².
  • The patient must have increased albuminuria, which means a UACR (urine albumin-to-creatinine ratio) of 300 mg/g or higher.
  • If the patient is taking an ACE inhibitor or ARB (medications for blood pressure and kidney protection), they should have been on a stable and well-tolerated dose for at least 60 days before the study starts.
  • If the patient is taking SGLT2 (a type of medication for diabetes and kidney protection), they should have been on a stable and well-tolerated dose for at least 60 days before the study starts.
  • If the patient has a history of high blood pressure, they should be on stable treatment for at least 60 days before the study starts, and their blood pressure should be controlled according to the study investigator.

Who Cannot Join the Study?

  • Patients who are not diagnosed with Alport Syndrome cannot participate. Alport Syndrome is a genetic condition that affects the kidneys, ears, and eyes.
  • Patients who are not at risk of progression of Alport Syndrome cannot participate. This means the condition is not expected to get worse over time.
  • Patients who are younger than 18 years old cannot participate.
  • Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups like pregnant women, children, or people who cannot make decisions for themselves.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsmedizin Goettingen Goettingen Germany
Centre Hospitalier Universitaire De Bordeaux Bordeaux France

Other Sites

Site Name City Country Status
Fundacio Puigvert Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Hospital Universitario Fundacion Jimenez Diaz Madrid Spain
University Clinical Hospital Virgen De La Arrixaca Murcia Spain
Centre Hospitalier Universitaire De Montpellier Montpellier France
Hopital Beaujon Clichy France
Asuaqkpxzh Pckdlagl Henlssxd Dk Mnqdzrojy Marseille France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not recruiting
15.07.2024
Germany Germany
Not recruiting
15.07.2024
Spain Spain
Not recruiting
15.07.2024

Trial locations

Investigated drugs:

Vonafexor is being studied to see if it is safe and well-tolerated in patients with Alport syndrome who are at risk of disease progression. The trial aims to understand how patients respond to the medication both while they are taking it and after they stop.

Investigated diseases:

Alport Syndrome – Alport Syndrome is a genetic disorder that primarily affects the kidneys, ears, and eyes. It is characterized by progressive kidney disease, hearing loss, and eye abnormalities. The condition is caused by mutations in genes responsible for producing type IV collagen, a protein essential for the normal function of the basement membranes in these organs. Over time, individuals with Alport Syndrome may experience worsening kidney function, leading to chronic kidney disease. Hearing loss typically begins in childhood or adolescence and can progress to significant impairment. Eye changes may include anterior lenticonus and retinal flecks, which can affect vision.

Trial ID:
2023-509638-20-00
Protocol code:
EYP001-208
Trial Phase:
Therapeutic exploratory (Phase II)

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