Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)

NCT ID: NCT01521793

Last Updated: 2014-07-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 27 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-01-31
• Study Completion Date: 2014-06-30

Brief Summary

The purpose of this study is:

• To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects treated previously with a single 7-day course of QLT091001 in Study RET IRD 01
• To evaluate whether up to 3 additional courses of oral QLT091001 administered once daily for 7 days can maintain or improve visual function.

Conditions

LCA (Leber Congenital Amaurosis); RP (Retinitis Pigmentosa)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

QLT091001

oral QLT091001 administered once daily for 7 days

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Subjects with LCA or RP due to RPE65 or LRAT deficiency who received a 7-day treatment course of QLT091001 and completed the Day 30 visit in Study RET IRD 01
• Subjects who meet any one of the following criteria at least 1 month after start of the 7-day treatment course in Study RET IRD 01:

• no increase in GVF (in at least 1 eye): Follow-up GVF increased ≤20% from baseline or
• decrease in GVF (in at least 1 eye): Follow-up GVF decreased below the highest previous response by ˃20% or
• Considered a reasonable candidate for retreatment, based on the Investigator's discretion, in consultation with the independent DSMB and the Sponsor, based on regression or lack of response in other parameters of visual function (e.g., subjective reports of changes in color vision or adaptation to low light) but who do not meet other (GVF) criteria for study entry

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 liver failure, uncontrolled thyroid disease, hypersensitivity to retinoids, or hypervitaminosis A
• Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of screening

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

QLT Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sushanta Mallick

Role: STUDY_DIRECTOR

QLT Inc.

Locations

The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)

Chicago, Illinois, United States

Wilmer Eye Institute (Johns Hopkins University)

Baltimore, Maryland, 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

United States; Canada; Germany; Netherlands; United Kingdom

Other Identifiers

RET IRD 02

Identifier Type: -

Identifier Source: org_study_id