CompletedPhase IIInterventional

Bestrophin 1 treatment trial on the effectiveness of Ravicti

This clinical trial is investigating whether an existing medicine called Ravicti can help treat inherited eye conditions known as bestrophinopathies, including Best disease (BVMD) and autosomal recessive bestrophinopathy (ARB). In these conditions, a faulty protein prevents cells at the back of the eye from working properly, leading to progressive vision loss. Ravicti is already approved for a different condition, but research suggests it may also help this faulty eye protein work better. In this study, run by Manchester University NHS Foundation Trust, participants aged 18 to 65 will take Ravicti for seven days and a dummy medicine (placebo) for seven days, with a break in between. Researchers will measure the eye's natural electrical signals to see if Ravicti improves cell activity. The trial aims to find out if repurposing this medicine could become a future treatment option for genetic eye conditions.

At a glance

Status

Completed

Phase

Phase II

Sponsor

Manchester University NHS Foundation Trust

Enrolment target

Start

01 Mar 2024

Estimated completion

18 Jun 2025

Autosomal dominant best vitelliform macular dystrophy (BVMD) or autosomal recessive bestrophinopathy (ARB)

What is this study about?

Bestrophinopathies are a group of rare, inherited eye conditions that cause gradual sight loss. They include conditions such as Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB). In a healthy eye, a protein called bestrophin works like a tiny gatekeeper, helping essential minerals move in and out of cells in the retina at the back of the eye. When this protein is faulty, the cells cannot work properly, which gradually damages the light-sensing cells needed for clear vision.

Currently, there are very few treatments that address the root cause of these conditions. This study is testing a medicine called Ravicti. While Ravicti is already used safely within the NHS for an unrelated metabolic condition, laboratory research has shown that it might also help faulty bestrophin proteins work more effectively.

Researchers want to see if taking Ravicti can temporarily restore protein activity in patients. To measure this, doctors use a safe, routine test called an electrooculogram (EOG). This test checks the natural electrical responses in the eye, which show how well the bestrophin protein is performing. If the test results improve while taking Ravicti, it could prove that the medicine is helping the cells function again.

By testing a medicine that is already approved for human use, doctors hope to speed up the discovery of treatments that could one day slow down or prevent vision loss for people living with genetic eye diseases.

Key takeaways

The trial tests whether the drug Ravicti can improve cell function in rare inherited eye conditions (bestrophinopathies).
It is open to adults aged 18 to 65 with a confirmed diagnosis of BVMD (Best disease) or ARB.
Participants take Ravicti for one week and a dummy medicine (placebo) for one week, with a three-week break in between.
Non-invasive electrical eye tests (EOG) will measure whether the medicine helps eye cells work better.
Taking part is voluntary, and you can withdraw at any point without affecting your routine NHS care.

Who may be eligible?

You may be able to join this study if you are between 18 and 65 years old and have a confirmed genetic and clinical diagnosis of an inherited bestrophin eye condition (such as Best disease or ARB). You must have some remaining vision (better than only seeing hand movements) and be able to speak and understand English so you can follow the instructions and give informed consent.

You will not be able to take part if you are pregnant, planning a pregnancy, or breastfeeding. The trial is also not suitable for people with liver disease, serious gut absorption problems, or certain metabolic disorders (such as urea cycle disorders).

In addition, you cannot join if you have taken part in another clinical drug trial within the past 12 weeks, or if you take specific prescription medicines that could interact with Ravicti, including probenecid, certain strong sedatives, or particular immune-system drugs. The study team can review your current medications to see if they are safe to use during the trial.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

I am aged between 18 and 65 years old.
I have a confirmed clinical and genetic diagnosis of Best disease (BVMD) or ARB.
My eyesight is better than only being able to see hand movements.
I am not pregnant, planning a pregnancy, or breastfeeding.
I do not have liver disease or serious gut absorption problems.
I have not taken part in another clinical drug trial in the last 12 weeks.

Answer every question to see your result.

What does participation involve?

Taking part involves taking the study medicine (Ravicti) by mouth for 7 days, followed by a 21-day rest period with no medication, and taking a dummy medicine (placebo) for 7 days. Because this is a 'crossover' trial, every participant tests both the real medicine and the placebo at different times, but neither you nor the doctors will know which one you are taking first.

You will need to visit the clinic at Manchester University NHS Foundation Trust for eye examinations and safety checks. These include standard eye tests that record electrical activity in the eye (EOG and ERG), a routine blood test to check your liver health, and a urine pregnancy test for women. A doctor is on call 24 hours a day if you feel unwell or have questions about taking the study drug.

Potential risks and benefits

Because Ravicti is an approved medicine, its effects are well understood, and any side effects are typically mild and temporary. The study eye tests are non-invasive and safe, though having a blood sample taken can cause minor discomfort or brief bruising. Taking part may not directly improve your eyesight in the long term, but the results could help scientists develop new treatments for genetic sight loss in the future. You are free to leave the trial at any time without giving a reason, and your regular NHS eye care will not be affected.

Locations (1)

St Mary's Hospital
Approximate
Manchester, England

Common questions

Will taking Ravicti cure my sight loss?

No, this trial is not a cure. It is an early-stage study designed to see if the medicine can temporarily help faulty eye cells work better. Taking part is unlikely to give you permanent vision improvement.

What is a placebo and why is it used?

A placebo looks just like the real medicine but contains no active ingredients. Comparing Ravicti against a placebo helps researchers know whether any changes in your eye tests are truly caused by the drug.

Does the electrooculogram (EOG) eye test hurt?

No. The EOG is a safe, painless test that uses small sticky sensors placed on the skin around your eyes to record natural electrical signals while you look at lights.

What happens if I experience side effects during the study?

You will be given a 24-hour phone number for the study medical team. If you feel unwell, a doctor can advise you and, if necessary, immediately find out which treatment you were taking to ensure you receive the right care.

Do I have to travel to Manchester to take part?

Yes, all study visits, health checks, and specialised eye tests are conducted at Manchester University NHS Foundation Trust.

How to find out more

Eva Lenassi

eva.lenassi@mft.nhs.uk +44 (0)161 276 1234 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.