Better – Bestrophin 1 treatment trial on the effectiveness of Ravicti

Ravicti as therapy for bestrophinopathies: a double-blind crossover randomized controlled trial

  • Hospital: Manchester Royal Eye Hospital – Manchester Centre for Genomic Medicine
  • Study Type: CTIMP – Clinical Trial of an Investigational Medicinal Product
  • Funder: National Institute for Health and Care Excellence (NIHR)


This study will investigate whether the drug Ravicti is an effective 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 facilitates 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 measured in the clinic by the electrooculogram (EOG) which is characteristically abnormal in bestrophinopathy patients. Ravicti is already approved for an unrelated condition, but is also known to improve the function of 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 to establish a starting value before dosing with Ravicti for seven days before having another EOG. 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.

Participant Group

Patients with clinical diagnosis and molecular confirmation of autosomal dominant Best vitelliform macular dystrophy (BVMD) or autosomal recessive bestrophinopathy (ARB).

Participant Approach

Patients appearing eligible will be approached by the research team, PI or Co-I with the participant information leaflet and consent forms. The initial contact may be made by telephone or post, as deemed appropriate by the clinician.

Study open date and length

Opened: 25 April 2024

Expected end date: April 2025


Pending – study ongoing

Contact Details

Email address:

Telephone number: 0161 701 4914


  • IRAS Number 1006836