- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01521793
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
July 25, 2014 updated by: QLT Inc.
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.
Study Overview
Status
Completed
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
27
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Quebec
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Montreal, Quebec, Canada
- Montreal Children's hospital, Mcgill University Health Centre
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Tubingen, Germany
- Institute for Ophthalmic Research, University of Tübingen
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Rotterdam, Netherlands
- The Rotterdam Eye Hospital
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London, United Kingdom, EC1 V2PD
- Moorefield Eye Hospital
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Illinois
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Chicago, Illinois, United States, 60608
- The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)
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Maryland
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Baltimore, Maryland, United States, 21287
- Wilmer Eye Institute (Johns Hopkins University)
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
5 years to 65 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Visual field
Time Frame: 12 months
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12 months
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs
Time Frame: 12 months
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12 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Sushanta Mallick, QLT Inc.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
January 1, 2012
Primary Completion (Actual)
May 1, 2014
Study Completion (Actual)
June 1, 2014
Study Registration Dates
First Submitted
January 6, 2012
First Submitted That Met QC Criteria
January 30, 2012
First Posted (Estimate)
January 31, 2012
Study Record Updates
Last Update Posted (Estimate)
July 28, 2014
Last Update Submitted That Met QC Criteria
July 25, 2014
Last Verified
July 1, 2014
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Nervous System Diseases
- Eye Diseases
- Neurologic Manifestations
- Retinal Degeneration
- Retinal Diseases
- Genetic Diseases, Inborn
- Eye Diseases, Hereditary
- Retinal Dystrophies
- Sensation Disorders
- Vision Disorders
- Retinitis
- Retinitis Pigmentosa
- Blindness
- Leber Congenital Amaurosis
- Physiological Effects of Drugs
- Antineoplastic Agents
- Immunologic Factors
- Protective Agents
- Adjuvants, Immunologic
- Anticarcinogenic Agents
- Retinol acetate
Other Study ID Numbers
- RET IRD 02
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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