RET NAT 01 - Natural History Study in Inherited Retinal Disease
Research type
Research Study
Full title
Retrospective, Uncontrolled, Multicenter, Case History Study to Determine the Natural History of Visual Function in Subjects with Inherited Retinal Disease (IRD) Caused by Inherited Mutation of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)
IRAS ID
191234
Contact name
Michel Michaelides
Contact email
Sponsor organisation
QLT Inc.
Clinicaltrials.gov Identifier
NCT02575430, Clinicaltrials.gov listing
Duration of Study in the UK
0 years, 0 months, 31 days
Research summary
Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) are rare inherited diseases of the eye which result in severe visual impairment and blindness. There are many different gene mutations that cause LCA and RP. The genes that are of interest for this research study affect the production (in the eye) of a substance called 11-cis-retinal, which is needed for normal vision. Two such genes are called RPE65 and LRAT. Mutations of these genes slow down or prevent the production of 11-cis-retinal causing loss of visual function and/or blindness.
The Sponsor of this study, QLT Inc., is developing a drug called QLT091001 to treat LCA and RP due to mutations in RPE65 and LRAT. This study does not involve treatment with the drug or having any procedures performed. It is limited to the collection of the results of vision tests that patients with mutations in RPE65 and LRAT have already done.
The purpose of this study is to learn more about how having LCA or RP affects people’s vision over time. This study will include up to 60 patients with LCA or RP caused by mutations in RPE65 and LRAT, some of which have previously participated in studies of QLT091001. The study will be running in Europe and North America.
Once the data is analysed, the vision change in untreated patients can be compared to vision changes in patients who were treated in other studies. This will help researchers understand whether a treatment is working.
REC name
London - Surrey Research Ethics Committee
REC reference
15/LO/2136
Date of REC Opinion
4 Dec 2015
REC opinion
Favourable Opinion