- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01014052
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
May 13, 2013 updated by: QLT Inc.
Phase 1b Study to Evaluate QLT091001 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)
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)
Study Overview
Status
Completed
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
32
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, H3H 1P3
- 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 for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)
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Maryland
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Baltimore, Maryland, United States, 21287
- Wilmer Eye Institute (Johns Hopkins University)
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Scheie Eye Institute
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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 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
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 |
---|---|
Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events
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.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- Scholl HP, Moore AT, Koenekoop RK, Wen Y, Fishman GA, van den Born LI, Bittner A, Bowles K, Fletcher EC, Collison FT, Dagnelie G, Degli Eposti S, Michaelides M, Saperstein DA, Schuchard RA, Barnes C, Zein W, Zobor D, Birch DG, Mendola JD, Zrenner E; RET IRD 01 Study Group. Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT). PLoS One. 2015 Dec 10;10(12):e0143846. doi: 10.1371/journal.pone.0143846. eCollection 2015.
- Wen Y, Birch DG. Outer Segment Thickness Predicts Visual Field Response to QLT091001 in Patients with RPE65 or LRAT Mutations. Transl Vis Sci Technol. 2015 Oct 1;4(5):8. doi: 10.1167/tvst.4.5.8. eCollection 2015 Oct.
- Koenekoop RK, Sui R, Sallum J, van den Born LI, Ajlan R, Khan A, den Hollander AI, Cremers FP, Mendola JD, Bittner AK, Dagnelie G, Schuchard RA, Saperstein DA. Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial. Lancet. 2014 Oct 25;384(9953):1513-20. doi: 10.1016/S0140-6736(14)60153-7. Epub 2014 Jul 13.
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
November 1, 2009
Primary Completion (Actual)
August 1, 2012
Study Completion (Actual)
August 1, 2012
Study Registration Dates
First Submitted
November 12, 2009
First Submitted That Met QC Criteria
November 13, 2009
First Posted (Estimate)
November 16, 2009
Study Record Updates
Last Update Posted (Estimate)
May 14, 2013
Last Update Submitted That Met QC Criteria
May 13, 2013
Last Verified
May 1, 2013
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 01
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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