Open-Label Extension Assessing Long Term Safety and Efficacy of ISIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)


About this study

This study evaluates the safety and tolerability of extended dosing with ISIS-TTR Rx 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:

  • Satisfactory completion of dosing & efficacy assessments in ISIS 420915-CS2

Exclusion Criteria:

  • Any new condition or worsening of existing condition that could make the patient unsuitable for participation, or interfere with the patient participating in and/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:

Morie Gertz M.D.

(507) 284-5096

More information


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