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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 has been completed.
Information provided by (Responsible Party):
QLT Inc. Identifier:
First received: November 12, 2009
Last updated: May 13, 2013
Last verified: May 2013

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: 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)

Resource links provided by NLM:

Further study details as provided by QLT Inc.:

Primary Outcome Measures:
  • Visual Field [ Time Frame: 12 months ]

Secondary Outcome Measures:
  • Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 12 months ]

Enrollment: 32
Study Start Date: November 2009
Study Completion Date: August 2012
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: QLT091001
    oral QLT091001 administered once daily for 7 days

Ages Eligible for Study:   5 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01014052

United States, Illinois
The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)
Chicago, Illinois, United States, 60608
United States, Maryland
Wilmer Eye Institute (Johns Hopkins University)
Baltimore, Maryland, United States, 21287
United States, Pennsylvania
Scheie Eye Institute
Philadelphia, Pennsylvania, United States, 19104
Canada, Quebec
Montreal Children's Hospital, McGill University Health Centre
Montreal, Quebec, Canada, H3H 1P3
Institute for Ophthalmic Research, University of Tubingen
Tubingen, Germany
The Rotterdam Eye Hospital
Rotterdam, Netherlands
United Kingdom
Moorefield Eye Hospital
London, United Kingdom, EC1 V2PD
Sponsors and Collaborators
QLT Inc.
Study Director: Sushanta Mallick QLT Inc.
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: QLT Inc. Identifier: NCT01014052     History of Changes
Other Study ID Numbers: RET IRD 01
Study First Received: November 12, 2009
Last Updated: May 13, 2013

Additional relevant MeSH terms:
Leber Congenital Amaurosis
Retinitis Pigmentosa
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
Vitamin A
Retinol acetate
Growth Substances
Physiological Effects of Drugs
Adjuvants, Immunologic
Immunologic Factors
Anticarcinogenic Agents
Protective Agents
Antineoplastic Agents processed this record on September 21, 2017