A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Pol… (NCT04201418) | Clinical Trial Compass
CompletedNot Applicable
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
United States67 participantsStarted 2019-12-18
Plain-language summary
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* 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.
Exclusion Criteria:
* 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
* Prior major organ transplant
* Previously received patisiran
* Previous treatment with a TTR silencing therapy
What they're measuring
1
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline