Classic Galactosemia Clinical Trial
Official title:
Does Arginine Enhance Galactose Oxidative Capacity in Classic Galactosemia: A Pilot Study
Rationale: Classic galactosemia is a rare inherited metabolic disease that presents in
neonatal patients with a life-threatening multi-organ toxic syndrome. Although the current
standard of care - a galactose-restricted diet - quickly relieves the severe neonatal
clinical picture, it fails to prevent brain and gonadal sequelae. There is a need for new
therapeutic strategies.
As arginine is an amino acid that is therapeutically widely used with no side effects
described, we propose to use it in a pilot-clinical study. We aim to evaluate the effects of
arginine in classic galactosemia patients, in order to determine its potential therapeutic
role in this disease.
Objective: To evaluate the possible effect of arginine on the whole body galactose oxidative
capacity in classic galactosemia patients.
Study design: Interventional pilot-clinical study with pre-post single arm design.
Study population: We aim to include 5 classic galactosemia adult patients homozygous for the
p.Q188R mutation.
Intervention: All participants will receive arginine in the form of Asparten ® (arginine
aspartate) during 1 month, by oral administration.
The main study parameter is whole body galactose galactose oxidative capacity.
n/a
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