PQ-110-005: Study to evaluate sepofarsen in pediatric LCA10 patients

• Research type

  Research Study

• Full title

  An open-label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects of Age 3 to <8 Years with Leber Congenital Amaurosis Type 10 (LCA10) due to the c.2991+1655A>G (p.Cys998X) mutation

• IRAS ID

  1003428

• Contact name

  Michel Michaelides

• Contact email

  michel.michaelides@nhs.net

• Eudract number

  2020-000535-45

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  20/NW/0382

• Date of REC Opinion

  16 Dec 2020

• REC opinion

  Further Information Favourable Opinion