A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients with Polyneuropathy of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with a V122I or T60A Mutation

  • Clinical Trial Information

    Trial Contact: Peisner, Pamela

    Trial Phone: 321.843.1037

  • IRB No: 20.110.07

    Protocol Abbrev: ALN-TTR02-012

    Principal Investigator: Shahid Qamar, MD

    Age Group: Adult

    Treatment: ONPATTRO (patisiran, ALN-TTR02) Lipid Complex injection, for intravenous use ID: 04201418

  • Objective

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

  • Key Eligibility

    1. 18 years or older.
    2. Diagnosed with hATTR amyloidosis with polyneuropathy, with a documented V122I or
    T60A mutation.
    3. PND score of I-IIIB at the baseline visit (see Section 10.1).