Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
This trial is active, not recruiting.
|Condition||retinitis pigmentosa (rp)|
|Start date||February 2012|
|End date||June 2014|
|Trial size||5 participants|
|Trial identifier||NCT01543906, RET RP 01|
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.
|United States||No locations recruiting|
|Other countries||No locations recruiting|
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
oral QLT091001 administered once daily for 7 days
time frame: 12 months
Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs
time frame: 12 months
Male or female participants from 18 years up to 70 years old.
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.
|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)|
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