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

• ClinicalTrials.gov Identifier: NCT01521793
• Recruitment Status: Completed
• First Posted: January 31, 2012
• Last Update Posted: July 28, 2014
• Sponsor: QLT Inc.
• Information provided by (Responsible Party): QLT Inc.

Tracking Information

• First Submitted Date: January 6, 2012
• First Posted Date: January 31, 2012
• Last Update Posted Date: July 28, 2014
• Study Start Date: January 2012
• Actual Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: February 28, 2012): Visual field [ Time Frame: 12 months ]

Original Primary Outcome Measures (submitted: January 30, 2012)

Visual field [ Time Frame: 12 months ]

Percent changed from baseline

• Current Secondary Outcome Measures (submitted: February 28, 2012): Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs [ Time Frame: 12 months ]

Original Secondary Outcome Measures (submitted: January 30, 2012)

• Best corrected visual acuity [ Time Frame: 12 months ]
  Scores and changes from baseline
• Color Vision [ Time Frame: 12 months ]
  Change from baseline
• Retinal thickness and fluorescence [ Time Frame: 12 months ]
  Change from baseline

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

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

Brief Summary

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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• LCA (Leber Congenital Amaurosis)
• RP (Retinitis Pigmentosa)

Intervention

Drug: QLT091001

oral QLT091001 administered once daily for 7 days

• Study Arms: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: July 25, 2014): 27
• Original Estimated Enrollment (submitted: January 30, 2012): 28
• Actual Study Completion Date: June 2014
• Actual Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 5 Years to 65 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, Germany, Netherlands, United Kingdom, United States

Administrative Information

• NCT Number: NCT01521793
• Other Study ID Numbers: RET IRD 02
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: QLT Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: QLT Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Sushanta Mallick; QLT Inc.

• PRS Account: QLT Inc.
• Verification Date: July 2014