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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
QLT Inc.
ClinicalTrials.gov Identifier:
NCT01521793
First received: January 6, 2012
Last updated: July 25, 2014
Last verified: July 2014
  Purpose

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 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: 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)

Resource links provided by NLM:


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:
  • Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Enrollment: 27
Study Start Date: January 2012
Study Completion Date: June 2014
Primary Completion Date: May 2014 (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 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
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 ClinicalTrials.gov identifier: NCT01521793

Locations
United States, Illinois
The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)
Chicago, Illinois, United States, 60608
United States, Maryland
Wilmer Eye Institute (Johns Hopkins University)
Baltimore, Maryland, United States, 21287
Canada, Quebec
Montreal Children's Hospital, McGill University Health Centre
Montreal, Quebec, Canada
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: NCT01521793     History of Changes
Other Study ID Numbers: RET IRD 02
Study First Received: January 6, 2012
Last Updated: July 25, 2014
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 August 28, 2014