Blood neurofilament light as a potential endpoint in Phase 2 studies in MS.

ABSTRACT:

Objectives

To assess whether neurofilament light chain (NfL) could serve as an informative endpoint in Phase 2 studies in patients with relapsing-remitting multiple sclerosis (RRMS) and estimate the sample size requirements with NfL as the primary endpoint.

Methods

Using data from the Phase 3 FREEDOMS study, we evaluated correlation of NfL at Month 6 with 2-year outcomes: relapses, confirmed disability worsening (CDW), new or enlarging T2 lesions (active lesions), and brain volume loss (BVL). We compared the proportion of treatment effect (PTE) on 2-year relapses and BVL explained by 6-month log-transformed NfL levels with the PTE explained by the number of active lesions over 6 months. We estimated sample size requirements for different treatment effects.

Results

At

SUBMITTER: Sormani MP 

PROVIDER: S-EPMC6562031 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature