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

Tracking Information

First Received Date ^ICMJE

November 12, 2009

Last Updated Date

May 13, 2013

Start Date ^ICMJE

November 2009

Primary Completion Date

August 2012 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Visual Field [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

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 ]

Change History

Complete list of historical versions of study NCT01014052 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Original Secondary Outcome Measures ^ICMJE

Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 14 days ] [ Designated as safety issue: Yes ]

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

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

Phase 1

Study Design ^ICMJE

Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Arm (s)

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

Enrollment ^ICMJE

Completion Date

August 2012

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

Gender

Both

Ages

5 Years to 65 Years (Child, Adult)

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Listed Location Countries ^ICMJE

United States, Canada, Germany, Netherlands, United Kingdom

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT01014052

Other Study ID Numbers ^ICMJE

RET IRD 01

Has Data Monitoring Committee

Plan to Share Data

Not Provided

IPD Description

Not Provided

Responsible Party

QLT Inc.

Study Sponsor ^ICMJE

QLT Inc.

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Director:

Sushanta Mallick

QLT Inc.

Information Provided By

QLT Inc.

Verification Date

May 2013

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

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