Overview

This trial is active, not recruiting.

Condition retinitis pigmentosa (rp)
Treatment qlt091001
Phase phase 1
Sponsor QLT Inc.
Start date February 2012
End date June 2014
Trial size 5 participants
Trial identifier NCT01543906, RET RP 01

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
oral QLT091001 administered once daily for 7 days
qlt091001
oral QLT091001 administered once daily for 7 days

Primary Outcomes

Measure
Visual field
time frame: 12 months

Secondary Outcomes

Measure
Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs
time frame: 12 months

Eligibility Criteria

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.

Additional Information

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)
Trial information was received from ClinicalTrials.gov and was last updated in March 2014.
Information provided to ClinicalTrials.gov by QLT Inc..