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

Sponsor

QLT Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT01521793

Collaborator

(none)

Enrollment

Locations

Duration (Months)

4.5

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
LCA (Leber Congenital Amaurosis) RP (Retinitis Pigmentosa)	Drug: QLT091001	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

27 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin: Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01)

Study Start Date :

Jan 1, 2012

Actual Primary Completion Date :

May 1, 2014

Actual Study Completion Date :

Jun 1, 2014

Outcome Measures

Primary Outcome Measures

Visual field [12 months]

Secondary Outcome Measures

Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs [12 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)	Chicago	Illinois	United States	60608
2	Wilmer Eye Institute (Johns Hopkins University)	Baltimore	Maryland	United States	21287
3	Montreal Children's Hospital, McGill University Health Centre	Montreal	Quebec	Canada
4	Institute for Ophthalmic Research, University of Tubingen	Tubingen		Germany
5	The Rotterdam Eye Hospital	Rotterdam		Netherlands
6	Moorefield Eye Hospital	London		United Kingdom	EC1 V2PD