NCT01521793

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2012

Typical duration for phase_1

Geographic Reach
5 countries

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2012

Completed
5 days until next milestone

First Submitted

Initial submission to the registry

January 6, 2012

Completed
25 days until next milestone

First Posted

Study publicly available on registry

January 31, 2012

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2014

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2014

Completed
Last Updated

July 28, 2014

Status Verified

July 1, 2014

Enrollment Period

2.3 years

First QC Date

January 6, 2012

Last Update Submit

July 25, 2014

Conditions

LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)

Outcome Measures

Primary Outcomes (1)

  • Visual field

    12 months

Secondary Outcomes (1)

  • Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs

    12 months

Interventions

QLT091001DRUG

oral QLT091001 administered once daily for 7 days

Eligibility Criteria

Age5 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)

Chicago, Illinois, 60608, United States

Location

Wilmer Eye Institute (Johns Hopkins University)

Baltimore, Maryland, 21287, United States

Location

Montreal Children's Hospital, McGill University Health Centre

Montreal, Quebec, Canada

Location

Institute for Ophthalmic Research, University of Tubingen

Tübingen, Germany

Location

The Rotterdam Eye Hospital

Rotterdam, Netherlands

Location

Moorefield Eye Hospital

London, EC1 V2PD, United Kingdom

Location

MeSH Terms

Conditions

Leber Congenital AmaurosisRetinitis Pigmentosa

Interventions

retinol acetate

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DiseasesRetinal DystrophiesRetinal DegenerationGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Sushanta Mallick

    QLT Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 6, 2012

First Posted

January 31, 2012

Study Start

January 1, 2012

Primary Completion

May 1, 2014

Study Completion

June 1, 2014

Last Updated

July 28, 2014

Record last verified: 2014-07

Locations

NL(1)GB(1)US(2)CA(1)DE(1)