BEstrophin 1 Treatment Trial on the Effectiveness of Ravicti (BETTER trial)
Research type
Research Study
Full title
Ravicti as therapy for bestrophinopathies: a double-blind crossover randomized controlled trial
IRAS ID
1006836
Contact name
Eva Lenassi
Contact email
Sponsor organisation
Manchester University Hospitals NHS Foundation Trust
ISRCTN Number
ISRCTN15509883
Research summary
This study will investigate whether the drug Ravicti is a therapeutic treatment for a group of inherited eye conditions called bestrophinopathies.
Bestrophinopathies result from faults in bestrophin, a protein in the pigmented layer of cells at the back of the eye that facilitate the passage of chloride ions into the cells. The faulty bestrophin reduces the flow of chloride ions, preventing the normal function of the pigmented cells. This in turn disrupts the normal function of the adjacent light-detecting cells, so causing sight loss. Bestrophin activity is easily measured in the clinic by the electrooculogram (EOG) which is characteristically abnormal in bestrophinopathy patients. In this study the EOG is a proxy measure of bestrophin function.
Ravicti is an approved drug for an unrelated condition, but is also known to improve the function of faulty proteins, including bestrophin. This study will test whether giving Ravicti to bestrophinopathy patients increases the function of their faulty bestrophin protein by measuring whether their EOG improves.
We will first measure the EOG in trial participants to establish a starting value before dosing them with Ravicti for 7 days. We will then measure the EOG again to see if the EOG improves. Patients will then be Ravicti-free for 21 days before measuring the EOG again to see whether it returns to the starting value. Each patient will also undergo the same schedule using a placebo to determine whether any improvement in the EOG is due to Ravicti or is a coincidence resulting from just being part of the study.REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
23/NE/0202
Date of REC Opinion
30 Nov 2023
REC opinion
Further Information Favourable Opinion