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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.
Sponsor:
Information provided by (Responsible Party):
QLT Inc.
ClinicalTrials.gov Identifier:
NCT01014052
First received: November 12, 2009
Last updated: May 13, 2013
Last verified: May 2013
History of Changes
  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:

MedlinePlus related topics: Vitamin A
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 ]

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
  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

Locations
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
Germany
Institute for Ophthalmic Research, University of Tubingen
Tubingen, Germany
Netherlands
The Rotterdam Eye Hospital
Rotterdam, Netherlands
United Kingdom
Moorefield Eye Hospital
London, United Kingdom, EC1 V2PD
Sponsors and Collaborators
QLT Inc.
Investigators
Study Director: Sushanta Mallick 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: May 13, 2013
Health Authority: Canada: Health Canada
United States: Food and Drug Administration
Germany: Federal Institute for Drugs and Medical Devices
Netherlands: Dutch Health Care Inspectorate
United Kingdom: Medicines and Healthcare Products Regulatory Agency

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 June 17, 2013