Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA)… (NCT01014052) | Clinical Trial Compass
CompletedPhase 1
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
United States, Canada, Germany32 participantsStarted 2009-11
Plain-language 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)
Who can participate
Age range5 Years – 65 Years
SexALL
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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