Open label ISIS 420915-CS3

• Research type

  Research Study

• Full title

  An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP)

• IRAS ID

  176954

• Contact name

  Carol Whelan

• Contact email

  c.whelan@ucl.ac.uk

• Sponsor organisation

  Isis Pharmaceuticals, Inc

• Eudract number

  2013-004561-13

• Duration of Study in the UK

  3 years, 4 months, 14 days

• Research summary

  This is a Phase 3 study to assess the long­term safety and tolerability of an experimental drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy FAP (an inherited disease where nerve function is limited due to a buildup of protein deposits in peripheral nerves).
  Approximately 195 patients will take part from many centres worldwide. Eligible patients who have satisfactorily completed or participated in a previous study (named ISIS 420915­ CS2) with the same experimental drug, will receive 300mg of the study drug, ISIS 420915. Patients that had a dose reduction or schedule change in the previous study ISIS 420915­CS2 may continue with the same dose level or schedule in this study. Participants will be expected to be in the study for 78 weeks (18 months) during which time they will attend 8 clinic visits. During these visits the following study procedures will be performed: blood tests, urine tests, electrocardiogram, electroretinograms and several clinical examinations. Also to be completed are a series of questionnaires. All patients will receive supplemental doses of vitamin A.
  After completing the treatment period, patients will enter the 3 ­months post­ treatment evaluation period.

• REC name

  London - Chelsea Research Ethics Committee

• REC reference

  15/LO/1174

• Date of REC Opinion

  23 Jul 2015

• REC opinion

  Favourable Opinion