Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study.

ABSTRACT:

Background

Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of patisiran (ALN-TTR02), a small interfering RNA encapsulated within lipid nanoparticles, in patients with transthyretin-mediated familial amyloid polyneuropathy (FAP).

Methods

In this phase II study, patients with FAP were administered 2 intravenous infusions of patisiran at one of the following doses: 0.01 (n = 4), 0.05 (n = 3), 0.15 (n = 3), or 0.3 (n = 7) mg/kg every 4 weeks (Q4W), or 0.3 mg/kg (n = 12) every 3 weeks (Q3W).

Results

Of 29 patients in the intent-to-treat population, 26 completed the study. Administration

SUBMITTER: Suhr OB 

PROVIDER: S-EPMC4559363 | biostudies-literature | 2015 Sep

REPOSITORIES: biostudies-literature