A randomized, double-blind, placebo-controlled study evaluating Acetazolamide efficacy in ataxia in PMM2-CDG
PMM2 Acetazolamide Trial
What is the goal of the study?
Multicenter, randomized, double-blind, placebo-controlled trial to determine the efficacy of acetazolamide in improving ataxia in patients with PMM2-CDG. 6 Subjects at our site will be randomized to placebo/Acetazolamide on a 1:1 ratio for 6 months then crossed over to open label treatment on Acetazolamide.
Who can participate in the study?
Please contact the study team listed below to learn more.