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
Conditions:Ocular
Therapuetic Areas:Ophthalmology
Healthy:No
Age Range:Any
Updated:9/23/2012
Start Date:November 2009
End Date:February 2013
Contact:Medical Information
Email:medaffairs@qltinc.com
Phone:1-800-663-5486

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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
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