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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

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ClinicalTrials.gov Identifier: NCT01014052
Recruitment Status : Completed
First Posted : November 16, 2009
Last Update Posted : May 14, 2013
Sponsor:
Information provided by (Responsible Party):
QLT Inc.

Tracking Information
First Submitted Date  ICMJE November 12, 2009
First Posted Date  ICMJE November 16, 2009
Last Update Posted Date May 14, 2013
Study Start Date  ICMJE November 2009
Actual Primary Completion Date August 2012   (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: November 13, 2009)
Best-corrected visual acuity, color vision, visual fields, electroretinogram (ERG) and full-field stimulus threshold test (FST) [ Time Frame: 14 days ]
Change History Complete list of historical versions of study NCT01014052 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 16, 2011)
Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 12 months ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 13, 2009)
Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 14 days ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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
Official Title  ICMJE 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)
Brief Summary

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)
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 *

*   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: May 13, 2013)
32
Original Estimated Enrollment  ICMJE
 (submitted: November 13, 2009)
8
Actual Study Completion Date  ICMJE August 2012
Actual Primary Completion Date August 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
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 NCT01014052
Other Study ID Numbers  ICMJE RET IRD 01
Has Data Monitoring Committee No
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 May 2013

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