Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
| Status: | Completed |
|---|---|
| Conditions: | Ocular |
| Therapuetic Areas: | Ophthalmology |
| Healthy: | No |
| Age Range: | 5 - 65 |
| Updated: | 3/1/2014 |
| Start Date: | January 2012 |
| End Date: | March 2014 |
| Contact: | Medical Information |
| Email: | medaff@qltinc.com |
| Phone: | 1-877-764-3131 |
An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome 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) (Extension of Study RET IRD 01)
The purpose of this study is:
- To evaluate the safety of up to 3 additional courses of oral QLT091001 administered
once daily for 7 days in subjects treated previously with a single 7-day course of
QLT091001 in Study RET IRD 01
- To evaluate whether up to 3 additional courses of oral QLT091001 administered once
daily for 7 days can maintain or improve visual function.
- To evaluate the safety of up to 3 additional courses of oral QLT091001 administered
once daily for 7 days in subjects treated previously with a single 7-day course of
QLT091001 in Study RET IRD 01
- To evaluate whether up to 3 additional courses of oral QLT091001 administered once
daily for 7 days can maintain or improve visual function.
Inclusion Criteria:
- Subjects with LCA or RP due to RPE65 or LRAT deficiency who received a 7-day
treatment course of QLT091001 and completed the Day 30 visit in Study RET IRD 01
- Subjects who meet any one of the following criteria at least 1 month after start of
the 7-day treatment course in Study RET IRD 01:
- no increase in GVF (in at least 1 eye): Follow-up GVF increased ≤20% from
baseline or
- decrease in GVF (in at least 1 eye): Follow-up GVF decreased below the highest
previous response by ˃20% or
- Considered a reasonable candidate for retreatment, based on the Investigator's
discretion, in consultation with the independent DSMB and the Sponsor, based on
regression or lack of response in other parameters of visual function (e.g.,
subjective reports of changes in color vision or adaptation to low light) but
who do not meet other (GVF) criteria for study entry
Exclusion Criteria:
- 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 liver failure, uncontrolled thyroid disease, hypersensitivity to
retinoids, or hypervitaminosis A
- Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60
days of screening
We found this trial at
2
sites
Chicago, Illinois 60608
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