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

Retinitis Pigmentosa (RP) Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01543906
• Other study ID #: RET RP 01
• Status: Completed
• Phase: Phase 1
• First received: February 17, 2012
• Last updated: December 11, 2014
• Start date: February 2012
• Est. completion date: August 2014

Study information

• Verified date: December 2014
• Source: QLT Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health CanadaIreland: Irish Medicines Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: August 2014
• Est. primary completion date: July 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• Retinitis Pigmentosa (RP)

Intervention

Drug:
• QLT091001
oral QLT091001 administered once daily for 7 days

Locations

Country	Name	City	State
Canada	Montreal Children's Hospital, McGill University Health Centre	Montreal	Quebec
Ireland	Royal Victoria Eye and Ear Hospital	Dublin

Sponsors (1)

Lead Sponsor	Collaborator
QLT Inc.

Countries where clinical trial is conducted

Canada,  Ireland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Visual field		12 months	No
Secondary	Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs		12 months	Yes