Clinical trial NCT01521793: Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: LCA (Leber Congenital Amaurosis)
RP (Retinitis Pigmentosa)
Lead sponsor: QLT Inc.
Sponsor class: INDUSTRY
First posted: 2012-01-31
Enrollment: 27
Sex: ALL
Minimum age (years): 5
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Visual field
timeFrame: 12 months
Secondary outcomes:
measure: Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs
timeFrame: 12 months
Other outcomes:
Locations:
city: Chicago
country: United States
facility: The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)
state: Illinois
city: Baltimore
country: United States
facility: Wilmer Eye Institute (Johns Hopkins University)
state: Maryland
city: Montreal
country: Canada
facility: Montreal Children's Hospital, McGill University Health Centre
state: Quebec
city: Tubingen
country: Germany
facility: Institute for Ophthalmic Research, University of Tubingen
city: Rotterdam
country: Netherlands
facility: The Rotterdam Eye Hospital
city: London
country: United Kingdom
facility: Moorefield Eye Hospital
Study officials:
affiliation: QLT Inc.
name: Sushanta Mallick
role: STUDY_DIRECTOR
References: