A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

  • End date
    Jun 26, 2022
  • participants needed
  • sponsor
    Alnylam Pharmaceuticals
Updated on 26 November 2020
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To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Treatment Patisiran
Clinical Study IdentifierNCT04201418
SponsorAlnylam Pharmaceuticals
Last Modified on26 November 2020

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Inclusion Criteria

Is your age greater than or equal to 18 yrs?
Gender: Male or Female
Do you have any of these conditions: Hereditary Transthyretin-mediated (ATTRv) Amyloidosis or Peripheral Neuropathy or Hereditary Transthyretin-mediated (hATTR) Amyloidosis or Polyneuropa...?
Do you have any of these conditions: Peripheral Neuropathy or Polyneuropathy or Hereditary Transthyretin-mediated (ATTRv) Amyloidosis or Hereditary Transthyretin-mediated (hATTR) Amyloido...?
Do you have any of these conditions: Hereditary Transthyretin-mediated (ATTRv) Amyloidosis or Polyneuropathy or Peripheral Neuropathy or Hereditary Transthyretin-mediated (hATTR) Amyloido...?
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
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