RET IRD 02 - Extension study of Study RET IRD 01

• Research type

  Research Study

• Full 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)

• IRAS ID

  102596

• Contact name

  Anthony Moore

• Sponsor organisation

  QLT Inc.

• Eudract number

  2011-004214-42

• Clinicaltrials.gov Identifier

  NCT01521793

• Research summary

  This is an open-label study for subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) due to inherited deficiency of Retinal Pigment Epithelial (RPE65) or Licithin:Retinol Acyltransferase (LRAT) who previously completed follow-up through at least Day 30 in Study RET IRD 01, in which they received a single 7-day treatment course of QLT091001. In this retreatment study, subjects will receive up to 3 courses of once-daily oral dosing of QLT091001 (40 or 60 mg/m2) for 7 days. Retreatment is based on defined criteria. A minimum of 3 weeks is required between treatment courses. This study is ?½ To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects who have been treated previously with a single 7-day course of QLT091001 in Study RET IRD 01; ?½ To evaluate whether up to 3 additional courses of oral QLT091001 administered once daily for 7 days can maintain or improve visual function in subjects who have been treated previously with a single 7-day course of QLT091001 in Study RET IRD 01. In this study, initial treatment with 60 mg/m2 QLT091001 will be given only to subjects for whom there were no safety concerns after treatment with 40 mg/m2 QLT091001 and who did not appear to respond to the 40 mg/m2 dose of QLT091001 in Study RET IRD 01. Each subject will be in the study for up to 12 months after the last treatment course. Maximum follow-up for each subject is 36 months. The study will include up to 30 patients globally. In UK only adult patients diagnosed with RP will be enrolled. Approximately 13 patients will be enrolled in Europe from up to 3 sites. In USA, Canada and Netherlands children and adults with either LCA or RP will participate in the study.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  12/LO/0829

• Date of REC Opinion

  3 Sep 2012

• REC opinion

  Further Information Favourable Opinion