Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

• Status: Completed
• Trial end date: 2012-08-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is:

• to evaluate the safety of oral QLT091001

• to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations

• to evaluate duration of visual function improvement (if observed)

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: QLT Inc.
• Treatments: Retinol acetate; Vitamin A

Criteria

Inclusion Criteria:

• Subjects diagnosed with LCA or RP (with a mutation in either RPE65 or LRAT)

• Subjects with LCA must be 5-65 years of age

• Subjects with RP must be 18-65 years of age

• Subjects who have a "best-corrected" visual acuity of 3 letters or better (20/800 Snellen equivalent) or viable photoreceptors on OCT/FAF.

Exclusion Criteria:

• Subjects who are actively participating in an experimental therapy study or who have received experimental therapy within 60 days of Day 0.

• Subjects with any clinically important abnormal physical finding at Screening.

• Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane,® Soriatane®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.

• Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis.

• Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of screening