Progress Report 2015

Progress report on the L-serine HSAN1 Trial
by Florian Eichler, M.D.

There are 18 subjects currently enrolled in the two-year, double-blind, placebo-controlled trial studying the efficacy of L-serine in patients with HSAN1. This is lower than our original recruitment aim of 20. However, at the last meeting of the Data Safety Monitoring Board (DSMB) it was discussed that if two additional subjects could not be identified, then enrollment should be closed. Of the 18 subjects enrolled, all have completed their 48-week visit.

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. The investigators are blinded until the end of the study, so they currently cannot assess whether L-serine is more effective than placebo.

To date no adverse events have been deemed related to the study drug. To ensure data integrity and compliance with the study protocol, for which we receive our funding, we have regular DSMB meetings scheduled. During the last DSMB meeting the investigators also discussed an open label extension until the DSMB meeting following the last study visit.

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