The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)


About this study

The purpose of this study is to evaluate the safety and effectiveness of long-term dosing with (ALN-TTR02) patisiran in patients who have familial transthyretin mediated amyloidosis with polyneuropathy, and have completed a prior clinical study on patisiran.

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

  • Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug
  • Willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent

Exclusion Criteria

  • Any new or uncontrolled condition that could make the patient unsuitable for participation

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

Angela Dispenzieri, M.D.

Closed for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

More information


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