An Open-Label, Phase 1b Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)
We have completed a Phase 1b multi-center, open-label clinical proof-of-concept trial to evaluate the safety profile and effects of a single seven-day course of treatment with QLT091001 on various parameters of retinal function and quality of life in patients with LCA and autosomal recessive RP due to inherited mutations in RPE65 or LRAT. In the study, LCA and RP patients received daily oral doses of QLT091001 for seven days with post-treatment follow-up at regular intervals for as long as visual function or subjective improvements were observed or until the patient was enrolled in our retreatment study (RET IRD 02).
The study evaluated changes in several visual function parameters, including best-corrected visual acuity (VA) andvisual field (VF) over the duration of the treatment and post-treatment follow-up. Visual acuity measures the acuteness or clearness of an individual's central vision, expressed in the study as number of letters or number of lines read on a visual acuity eye chart. Visual field measures an individual's entire scope or width of (central + peripheral) vision, expressed in the study as retinal areas. Peripheral vision is important in day-to-day mobility, whereas central vision reflects the ability to read and do fine vision work. Various medical conditions such as LCA, RP and glaucoma are characterized by debilitating loss of visual field.
Positive results from our Phase 1b clinical proof-of-concept study were reported for the 14 LCA patients in May and October 2011 and for the 18 early-onset RP patients in March 2012. In July 14, 2014, results from this study for LCA patients were first published online in The Lancet.
For more information, visit ClinicalTrials.gov.
Contact Information
QLT Medical Information
1-877-764-3131
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