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

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

Tracking Information
First Submitted Date  ICMJE January 6, 2012
First Posted Date  ICMJE January 31, 2012
Last Update Posted Date July 28, 2014
Study Start Date  ICMJE January 2012
Actual Primary Completion Date May 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 28, 2012)
Visual field [ Time Frame: 12 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 30, 2012)
Visual field [ Time Frame: 12 months ]
Percent changed from baseline
Change History Complete list of historical versions of study NCT01521793 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE
 (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  ICMJE Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • LCA (Leber Congenital Amaurosis)
  • RP (Retinitis Pigmentosa)
Intervention  ICMJE Drug: QLT091001
oral QLT091001 administered once daily for 7 days
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 25, 2014)
27
Original Estimated Enrollment  ICMJE
 (submitted: January 30, 2012)
28
Actual Study Completion Date  ICMJE June 2014
Actual Primary Completion Date May 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Germany,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01521793
Other Study ID Numbers  ICMJE RET IRD 02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party QLT Inc.
Study Sponsor  ICMJE QLT Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Sushanta Mallick QLT Inc.
PRS Account QLT Inc.
Verification Date July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP