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

LCA (Leber Congenital Amaurosis) Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01521793
• Other study ID #: RET IRD 02
• Status: Completed
• Phase: Phase 1
• First received: January 6, 2012
• Last updated: July 25, 2014
• Start date: January 2012
• Est. completion date: June 2014

Study information

• Verified date: July 2014
• Source: QLT Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health CanadaUnited States: Food and Drug AdministrationGermany: Federal Institute for Drugs and Medical DevicesNetherlands: Dutch Health Care InspectorateUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: June 2014
• Est. primary completion date: May 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years to 65 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

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

Intervention

Drug:
• QLT091001
oral QLT091001 administered once daily for 7 days

Locations

Country	Name	City	State
Canada	Montreal Children's Hospital, McGill University Health Centre	Montreal	Quebec
Germany	Institute for Ophthalmic Research, University of Tubingen	Tubingen
Netherlands	The Rotterdam Eye Hospital	Rotterdam
United Kingdom	Moorefield Eye Hospital	London
United States	Wilmer Eye Institute (Johns Hopkins University)	Baltimore	Maryland
United States	The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)	Chicago	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
QLT Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Visual field		12 months	No
Secondary	Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs		12 months	Yes