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

Phase 1b Study to Evaluate 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)

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)

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