Text Size

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

November 12, 2009
May 13, 2013
November 2009
August 2012   (final data collection date for primary outcome measure)
Visual Field [ Time Frame: 12 months ] [ Designated as safety issue: No ]
Best-corrected visual acuity, color vision, visual fields, electroretinogram (ERG) and full-field stimulus threshold test (FST) [ Time Frame: 14 days ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01014052 on ClinicalTrials.gov Archive Site
Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 14 days ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
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
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)

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)
Not Provided
Interventional
Phase 1
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • LCA (Leber Congenital Amaurosis)
  • RP (Retinitis Pigmentosa)
Drug: QLT091001
oral QLT091001 administered once daily for 7 days
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
32
August 2012
August 2012   (final data collection date for primary outcome measure)

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
Both
5 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada,   Germany,   Netherlands,   United Kingdom
 
NCT01014052
RET IRD 01
No
QLT Inc.
QLT Inc.
Not Provided
Study Director: Sushanta Mallick QLT Inc.
QLT Inc.
May 2013

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