2016 Progress Report

Progress report on the L-serine HSAN1 Trial

by Florian Eichler, M.D.  (with Kailey Joan Walsh)

There were 18 subjects enrolled in this two-year, double-blind, placebo-controlled trial studying the efficacy of L-serine in patients with HSAN1. The study was initially randomized to placebo versus study drug, and both the participants and the investigators were blinded as to who was on placebo and who was on the study drug. At week 48 all participants were switched over to L-serine, regardless of their randomization from the previous year.

At the last DSMB (Data and Safety Monitoring Board) meeting (May 18, 2015) it was recommended that an open-label extension be added to the protocol. This would allow the continued collection of outcomes data on the first 16 subjects from the time they complete the original study to the time that the last two subjects complete the study in February 2016. Upon the board’s suggestion and after further investigation, it was decided that an extension of one year would be added to the protocol. After the original 2-year period, subjects would be given the option of being re-consented for the open label extension. All consented subjects would then be treated with L-serine (400 mg/kg/d) for an additional year.

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