Clareo Health | A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Alnylam Pharmaceuticals
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
PND score of I-IIIB at baseline.
Exposure to commercial patisiran in one of the 3 cohorts:
* Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran.
* Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment.
* Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.
You may not be if
New York Heart Association (NYHA) heart failure classification ≥3
Karnofsky Performance Status (KPS) \<60%
Unstable congestive heart failure (CHF)
Known primary amyloidosis (AL) or leptomeningeal amyloidosis
