QLT091001 for Retinitis Pigmentosa in RET IRD 01 Amend. 8
Research type
Research Study
Full title
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)
IRAS ID
82391
Contact name
Anthony Moore
Sponsor organisation
QLT Inc.
Eudract number
2011-002055-33
Clinicaltrials.gov Identifier
Research summary
This is a study to investigate whether a new study drug, QLT091001, improves vision of patients with rare inherited retinal disorders caused by mutations in genes encoding visual cycle proteins Also it will evaluate any side effects associated with use of the drug.The study will include up to 28 patients globally. In Europe adult patients diagnosed with a condition called Retinitis Pigmentosa (RP) will be enrolled. Approximately 12 patients will be enrolled in Europe from up to 3 sites. In North America and Canada children and adults with either Leber Congential Amaurosis (LCA) or RP will participate in the study. Patients will undergo various ophthalmic and systemic assessments over a 3-week screening period to see if they are eligible for the study. Eligible patients enrolled in the study will receive a once-daily dose of study drug (QLT091001 40 mg/m2), a solution taken by mouth, for 7 consecutive days. Patients will be treated on an outpatient basis but will receive the study treatment in a research clinic under medical supervision for each day of treatment. Various assessments similar to those conducted at screening will be performed throughout the 7 days of treatment. During this time and for 7 days after treatment, patients must limit vigorous physical activity and will be instructed to follow specific dietary guidelines to avoid excessive vitamin A intake.Various assessments of retinal structure and function will be performed on both eyes at baseline and follow up visits. Follow-up visits will occur at Day 14, Day 30 and then continue every 2-3 months through to 12 months after treatment. Various safety assessments including analysis of blood and urine samples, heart monitoring, and physical examination will also be conducted..Additional follow-up visits will be scheduled at quarterly intervals thereafter at the Investigator's discretion.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
11/NE/0163
Date of REC Opinion
23 Aug 2011
REC opinion
Further Information Favourable Opinion