Back to resultsRepeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
Primary Purpose
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
QLT091001
Sponsored by
About this trial
This is an interventional treatment trial for LCA (Leber Congenital Amaurosis)
Eligibility Criteria
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
Sites / Locations
- The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)
- Wilmer Eye Institute (Johns Hopkins University)
- Montreal Children's Hospital, McGill University Health Centre
- Institute for Ophthalmic Research, University of Tubingen
- The Rotterdam Eye Hospital
- Moorefield Eye Hospital
Outcomes
Primary Outcome Measures
Visual field
Secondary Outcome Measures
Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01521793
Brief Title
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
Official Title
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)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2014
Overall Recruitment Status
Completed
Study Start Date
January 2012 (undefined)
Primary Completion Date
May 2014 (Actual)
Study Completion Date
June 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
QLT Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
27 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
QLT091001
Intervention Description
oral QLT091001 administered once daily for 7 days
Primary Outcome Measure Information:
Title
Visual field
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sushanta Mallick
Organizational Affiliation
QLT Inc.
Official's Role
Study Director
Facility Information:
Facility Name
The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60608
Country
United States
Facility Name
Wilmer Eye Institute (Johns Hopkins University)
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Montreal Children's Hospital, McGill University Health Centre
City
Montreal
State/Province
Quebec
Country
Canada
Facility Name
Institute for Ophthalmic Research, University of Tubingen
City
Tubingen
Country
Germany
Facility Name
The Rotterdam Eye Hospital
City
Rotterdam
Country
Netherlands
Facility Name
Moorefield Eye Hospital
City
London
ZIP/Postal Code
EC1 V2PD
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
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