Back to resultsOral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
Primary Purpose
Retinitis Pigmentosa (RP)
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
QLT091001
Sponsored by
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa (RP)
Eligibility Criteria
Inclusion Criteria:
- Subjects will have RP caused by an autosomal dominant mutation in RPE65, as diagnosed by an ocular geneticist or ophthalmologist.
- Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent) or visible photoreceptor outer segments on OCT/FAF.
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/Roaccutane® or Soriatane/Neotigason®) 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.
Sites / Locations
- Montreal Children's Hospital, McGill University Health Centre
- Royal Victoria Eye and Ear Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
QLT091001
Arm Description
oral QLT091001 administered once daily for 7 days
Outcomes
Primary Outcome Measures
Visual field
Secondary Outcome Measures
Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01543906
Brief Title
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
Official Title
An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
Study Type
Interventional
2. Study Status
Record Verification Date
December 2014
Overall Recruitment Status
Completed
Study Start Date
February 2012 (undefined)
Primary Completion Date
July 2014 (Actual)
Study Completion Date
August 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
QLT Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is:
To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day treatment with oral QLT091001.
To evaluate the safety of oral QLT091001 administered once daily for 7 days in RP subjects with an autosomal dominant mutation in RPE65.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa (RP)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Arm Title
QLT091001
Arm Type
Experimental
Arm Description
oral QLT091001 administered once daily for 7 days
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 results, ECG's and vital signs
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subjects will have RP caused by an autosomal dominant mutation in RPE65, as diagnosed by an ocular geneticist or ophthalmologist.
Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent) or visible photoreceptor outer segments on OCT/FAF.
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/Roaccutane® or Soriatane/Neotigason®) 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.
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
Montreal Children's Hospital, McGill University Health Centre
City
Montreal
State/Province
Quebec
Country
Canada
Facility Name
Royal Victoria Eye and Ear Hospital
City
Dublin
Country
Ireland
12. IPD Sharing Statement
Citations:
PubMed Identifier
32573764
Citation
Schwartz SG, Wang X, Chavis P, Kuriyan AE, Abariga SA. Vitamin A and fish oils for preventing the progression of retinitis pigmentosa. Cochrane Database Syst Rev. 2020 Jun 18;6(6):CD008428. doi: 10.1002/14651858.CD008428.pub3.
Results Reference
derived
Learn more about this trial
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
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