Oral QLT091001 in Retinitis Pigmentosa(RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
RP is genetically heterogeneous and can be inherited in an autosomal recessive (AR), autosomal dominant (AD), or X-linked manner, with rare digenic and mitochondrial forms. Previously, all reported mutations in RPE65 were associated with recessive RP or LCA. Recently, a dominant-acting mutation in RPE65 was reported. This form of RP is rare and has a milder disease presentation than the autosomal recessive form.
In order to investigate the safety, tolerability and efficacy of oral QLT091001 as a novel treatment for RP patients with an autosomal dominant mutation in RPE65, an open-label, Phase 1b, proof-of-concept study was conducted. The study evaluated the safety and treatment effects of a single course (once-daily for seven days) of oral 40 mg/m2 QLT091001 in five RP patients with an autosomal dominant mutation in RPE65. Visual field (VF) was assessed using Goldmann Visual Field (GVF) and visual acuity (VA) was assessed using best-corrected visual acuity (BCVA) at baseline and days 7, 14, 30 and 60 after treatment, then bimonthly for up to 12 months.
The study has been completed and results of the study suggest that QLT091001 can improve visual function in patients with autosomal dominant RP due to RPE65 mutations with a safety profile similar to that seen in the Phase 1b clinical trials in LCA and RP patients (IRD) with autosomal recessive mutations in RPE65 or LRAT.
For more information, visit ClinicalTrials.gov.
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