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More info
You can access this
clinical trial
if you have
Hereditary Sensory and Autonomic Neuropathy Type I
and you are
over 18
years old
1
2
This is a medium phase trial determining
the safety and dosage of the new treatment, as well as its efficacy.
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The purpose

In hereditary sensory and autonomic neuropathy type 1 (HSAN1) the investigators recently discovered the accumulation of two neurotoxic sphingolipids. It appears that these lipids arise as the mutant enzyme has a reduced affinity for its normal preferred substrate L-serine. The investigators now plan to perform a two year study of L-serine supplementation to correct the biochemistry and neurological disease in humans with HSAN1. In the course the investigators will also establish correlations between an existing neurological rating scale of sensory neuropathy and intraepidermal nerve fiber density. Funding Source - FDA OOPD

Provided treatments

  • Drug: L-serine
  • Drug: placebo

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT01733407. The sponsor of the trial is Massachusetts General Hospital and it is looking for 18 volunteers for the current phase.
Official trial title:
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy of L-Serine in Subjects With Hereditary Sensory Neuropathy Type 1