Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

Sponsor

QLT Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT01014052

Collaborator

(none)

Enrollment

Locations

Duration (Months)

4.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is:

to evaluate the safety of oral QLT091001
to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations
to evaluate duration of visual function improvement (if observed)

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

Study Design

Study Type:

Interventional

Actual Enrollment :

32 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Phase 1b Study to Evaluate QLT091001 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)

Study Start Date :

Nov 1, 2009

Actual Primary Completion Date :

Aug 1, 2012

Actual Study Completion Date :

Aug 1, 2012

Outcome Measures

Primary Outcome Measures

Visual Field [12 months]

Secondary Outcome Measures

Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [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 diagnosed with LCA or RP (with a mutation in either RPE65 or LRAT)
Subjects with LCA must be 5-65 years of age
Subjects with RP must be 18-65 years of age
Subjects who have a "best-corrected" visual acuity of 3 letters or better (20/800 Snellen equivalent) or viable photoreceptors on OCT/FAF.

Exclusion Criteria:

Subjects who are actively participating in an experimental therapy study or who have received experimental therapy within 60 days of Day 0.
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 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)	Chicago	Illinois	United States	60608
2	Wilmer Eye Institute (Johns Hopkins University)	Baltimore	Maryland	United States	21287
3	Scheie Eye Institute	Philadelphia	Pennsylvania	United States	19104
4	Montreal Children's Hospital, McGill University Health Centre	Montreal	Quebec	Canada	H3H 1P3
5	Institute for Ophthalmic Research, University of Tubingen	Tubingen		Germany
6	The Rotterdam Eye Hospital	Rotterdam		Netherlands
7	Moorefield Eye Hospital	London		United Kingdom	EC1 V2PD