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

This study is currently recruiting participants.

Verified April 2012 by QLT Inc.

First Received on November 12, 2009. Last Updated on April 5, 2012 History of Changes

Sponsor:	QLT Inc.
Information provided by (Responsible Party):	QLT Inc.
ClinicalTrials.gov Identifier:	NCT01014052

Purpose

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	Intervention	Phase
LCA (Leber Congenital Amaurosis) RP (Retinitis Pigmentosa)	Drug: QLT091001	Phase 1

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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)

Resource links provided by NLM:

Genetics Home Reference related topics: Leber congenital amaurosis X-linked juvenile retinoschisis Help Me Understand Genetics

MedlinePlus related topics: Vitamin A

Drug Information available for: Retinol Lecithin

U.S. FDA Resources

Further study details as provided by QLT Inc.:

Primary Outcome Measures:

Visual Field [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	28
Study Start Date:	November 2009
Estimated Study Completion Date:	February 2013
Estimated Primary Completion Date:	February 2013 (Final data collection date for primary outcome measure)

Intervention Details:

oral QLT091001 administered once daily for 7 days

Eligibility

Ages Eligible for Study:	5 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT01014052

Contacts

Contact: Medical Information

1-800-663-5486

medaffairs@qltinc.com

Locations

United States, Illinois
The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)	Recruiting
Chicago, Illinois, United States, 60608
Contact: Carol White 312-447-3254
Principal Investigator: Gerald Fishman, MD
United States, Maryland
Wilmer Eye Institute (Johns Hopkins University)	Recruiting
Baltimore, Maryland, United States, 21287
Contact: Kristen Bowles, O.D., M.S. 410-614-6908
Principal Investigator: Hendrik Scholl, MD, M.A.
United States, Pennsylvania
Scheie Eye Institute	Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Sharon Wolfe-Schwartz 215-662-9981
Principal Investigator: Samuel Jacobson, MD, PhD
Canada, Quebec
Montreal Children's Hospital, McGill University Health Centre	Recruiting
Montreal, Quebec, Canada, H3H 1P3
Contact: Eunice Esteban 514.412.4400 ext 22891 or 22530
Principal Investigator: Robert Koenekoop, MD, PhD
Germany
Institute for Ophthalmic Research, University of Tubingen	Recruiting
Tubingen, Germany
Contact: Eberhart Zrenner +49 70 71 298 4786
Principal Investigator: Eberhart Zrenner
Netherlands
The Rotterdam Eye Hospital	Recruiting
Rotterdam, Netherlands
Contact: L. Ingeborgh van den Born, MD, PhD +31 0 10 401777
Principal Investigator: L. Ingeborgh van den Born, MD, PhD
United Kingdom
Moorefield Eye Hospital	Recruiting
London, United Kingdom, EC1 V2PD
Contact: Isabelle Molden +44 0 20 756 62264
Principal Investigator: Tony Moore

Sponsors and Collaborators

QLT Inc.

Investigators

Study Director:

Suzanne Cadden

QLT Inc.

More Information

No publications provided

Responsible Party:	QLT Inc.
ClinicalTrials.gov Identifier:	NCT01014052 History of Changes
Other Study ID Numbers:	RET IRD 01
Study First Received:	November 12, 2009
Last Updated:	April 5, 2012
Health Authority:	Canada: Health Canada

Additional relevant MeSH terms:

Retinitis
Retinitis Pigmentosa
Leber Congenital Amaurosis
Blindness
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies

Retinal Degeneration
Genetic Diseases, Inborn
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms

ClinicalTrials.gov processed this record on May 23, 2012