Efficacy and Safety of ISIS-TTR Rx in Familial Amyloid Polyneuropathy


About this study

The purpose of this study is to evaluate the efficacy and safety of ISIS-TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Stage 1 and Stage 2 FAP patients with the following:
    • NIS score within protocol criteria
    • Documented transthyretin variant by genotyping
    • Documented amyloid deposit by biopsy
  • Females of child-bearing potential must use appropriate contraception and must be non-pregnant and non-lactating. Males engaged in relations of child-bearing potential must 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
  • Known monoclonal gammopathy of undetermined significance or multiple myeloma
  • Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease)
  • If previously treated with Vyndaqel®, must have discontinued treatment for 2 weeks prior to Study Day 1. If previously treated with Diflunisal, must 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 yr 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
  • Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Morie Gertz, M.D.

Closed for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office


More information


Publications are currently not available

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