About this study

To evaluate the effectiveness of Patisiran on ambulatory status in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation.

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:

  • 18 years or older.
  • Diagnosed with hATTR amyloidosis with polyneuropathy, with a documented V122I or T60A mutation.
  • PND score of I-IIIB at the baseline visit.
  • Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with Patisiran.
  • Willing and able to comply with the study requirements and to provide written informed consent.

Exclusion Criteria:

Disease-specific Conditions or History

  • New York Heart Association (NYHA) heart failure classification ≥ 3 at the baseline visit.
  • Have a Karnofsky Performance Status (KPS) of < 60% at the baseline visit.
  • Unstable congestive heart failure (CHF), including patients who require additional diuretics at the baseline visit to achieve optimal treatment of CHF, or patients with a heart failure related hospitalization in the 8 weeks prior to the baseline visit.
  • Any condition that in the opinion of the Investigator would interfere with the patient’s participation and completion of the study assessments in the 12-month duration of the study. This includes significant active and poorly controlled (unstable) cardiovascular disorders and untreated hypo- or hyperthyroidism.
  • Known primary amyloidosis (AL) or leptomeningeal amyloidosis.
  • Prior major organ transplant.
  • Planned major surgery, including orthotopic liver transplantation (OLT) during the study period.
  • Had a malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.

Prior Medication Use

  • Has previously received patisiran prior to enrollment in this study.
  • Has received treatment with a TTR silencing therapy in the past 6 months.
  • Participated in a clinical trial for any investigational agent in the past 6 months.

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

Jacksonville, Fla.

Mayo Clinic principal investigator

Elizabeth Mauricio, M.D.

Closed for enrollment

Contact information:

Amber Baskin CCRP

More information


Publications are currently not available

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