Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

• Status: Completed
• Trial end date: 2017-11-07
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ionis Pharmaceuticals, Inc.
• Collaborator: GlaxoSmithKline

Criteria

Inclusion Criteria:

• Stage 1 and Stage 2 FAP participants with the following:

1. NIS score within protocol criteria

2. Documented transthyretin variant by genotyping

3. Documented amyloid deposit by biopsy

• Females of child-bearing potential must use appropriate contraception and be non-pregnant and non-lactating. Males engaging in relations of child-bearing potential are to use appropriate contraception

Exclusion Criteria:

• Low Retinol level at screen

• Karnofsky performance status ≤50

• Poor Renal function

• Known type 1 or type 2 diabetes mellitus

• Other causes of sensorimotor or autonomic neuropathy (for example, autoimmune disease)

• If previously treated with Vyndaqel®, will need to have discontinued treatment for 2 weeks prior to Study Day 1. If previously treated with Diflunisal, will need to have discontinued treatment for 3 days prior to Study Day 1

• Previous treatment with any oligonucleotide or siRNA within 12 months of screening

• Prior liver transplant or anticipated liver transplant within 1 year of screening

• New York Heart Association (NYHA) functional classification of ≥3

• Acute Coronary Syndrome or major surgery within 3 months of screening

• Known Primary or Leptomeningeal Amyloidosis

• Anticipated survival less than 2 years

• Any other conditions in the opinion of the investigator which interfere with the participant participating in or completing the study