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

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)

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01521793

Enrollment

Registered

2012-01-31

Start date

2012-01-31

Completion date

2014-06-30

Last updated

2014-07-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

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.

Interventions

DRUGQLT091001

oral QLT091001 administered once daily for 7 days

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

5 Years to 65 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Visual field	12 months

Secondary

Measure	Time frame
Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs	12 months

Countries

Canada, Germany, Netherlands, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026