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

Completed

Conditions studied: Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy

In brief

To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Key facts

Study ID

NCT04201418

Run by

Alnylam Pharmaceuticals

People needed

67

Starts

2019-12-18

Expected to finish

2022-05-24

Last updated by the study team

2022-06-06

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify 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 qualify 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
• Prior major organ transplant
• Previously received patisiran
• Previous treatment with a TTR silencing therapy

Where it is running

• Clinical Trial Site — Los Angeles, California, United States
• Clinical Trial Site — San Diego, California, United States
• Clinical Trial Site — Aurora, Colorado, United States
• Clinical Trial Site — Farmington, Connecticut, United States
• Clinical Trial Site — Jacksonville, Florida, United States
• Clinical Trial Site — Tampa, Florida, United States
• Clinical Trial Site — Braselton, Georgia, United States
• Clinical Trial Site — Macon, Georgia, United States
• Clinical Trial Site — Chicago, Illinois, United States
• Clinical Trial Site — Chicago, Illinois, United States
• Clinical Trial Site — Boston, Massachusetts, United States
• Clinical Trial Site — Detroit, Michigan, United States
• Clinical Trial Site — Jackson, Mississippi, United States
• Clinical Trial Site — Kansas City, Missouri, United States
• Clinical Trial Site — Omaha, Nebraska, United States
• Clinical Trial Site — Chapel Hill, North Carolina, United States
• Clinical Trial Site — Charlotte, North Carolina, United States
• Clinical Trial Site — Columbus, Ohio, United States
• Clinical Trial Site — Allentown, Pennsylvania, United States
• Clinical Trial Site — Bethlehem, Pennsylvania, United States
• Clinical Trial Site — Lancaster, Pennsylvania, United States
• Clinical Trial Site — Pittsburgh, Pennsylvania, United States
• Clinical Trial Site — Pittsburgh, Pennsylvania, United States
• Clinical Trial Site — Germantown, Tennessee, United States
• Clinical Trial Site — Austin, Texas, United States

Full record on ClinicalTrials.gov

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