Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)

Phase 1

Completed

• Conditions: Retinitis Pigmentosa (RP)
• Interventions: Drug: QLT091001

• Registration Number: NCT01543906
• Lead Sponsor: QLT Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2012-02
• Study First Submitted: 2012-02-17
• Study First Submitted QC: 2012-03-01
• Study First Posted: 2012-03-05
• Primary Completion: 2014-07
• Study Completion: 2014-08
• Status Verified: 2014-12
• Last Update Submitted: 2014-12-11
• Last Update Posted: 2014-12-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
QLT091001	QLT091001	oral QLT091001 administered once daily for 7 days

Primary Outcome Measures

Name	Time	Method
Visual field	12 months

Secondary Outcome Measures

Name	Time	Method
Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs	12 months

Trial Locations

Locations (2)

Montreal Children's Hospital, McGill University Health Centre; 🇨🇦 Montreal, Quebec, Canada

Royal Victoria Eye and Ear Hospital; 🇮🇪 Dublin, Ireland