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
NCT ID: NCT01014052
Last Updated: 2013-05-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
32 participants
INTERVENTIONAL
2009-11-30
2012-08-31
Brief Summary
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* 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)
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Interventions
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QLT091001
oral QLT091001 administered once daily for 7 days
Eligibility Criteria
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Inclusion Criteria
* 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 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
5 Years
65 Years
ALL
No
Sponsors
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QLT Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Sushanta Mallick
Role: STUDY_DIRECTOR
QLT Inc.
Locations
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The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)
Chicago, Illinois, United States
Wilmer Eye Institute (Johns Hopkins University)
Baltimore, Maryland, United States
Scheie Eye Institute
Philadelphia, Pennsylvania, United States
Montreal Children's Hospital, McGill University Health Centre
Montreal, Quebec, Canada
Institute for Ophthalmic Research, University of Tubingen
Tübingen, , Germany
The Rotterdam Eye Hospital
Rotterdam, , Netherlands
Moorefield Eye Hospital
London, , United Kingdom
Countries
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References
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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.
Other Identifiers
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RET IRD 01
Identifier Type: -
Identifier Source: org_study_id
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