Feasibility of Huntington disease trials in the disease prodrome

Abstract

In this issue of Neurology ®, Rosas et al.1 present a phase II trial of creatine in Huntington disease (HD). This study is remarkable not only for the demonstration of safety and tolerability but also because the study design establishes the feasibility of clinical trials among premanifest HD individuals. Notably, the trial is undertaken in the disease prodrome, when the participants are clinically unaffected by HD, rather than in established disease. Furthermore, not all participants knew their mutation carrier status. The trial design defines methods to preserve confidentiality and overcomes the difficulty of studying HD at its earliest phase by avoiding the requirement to include only the genetically tested. The success of this study demonstrates that sufficient discriminant power can be obtained without the exclusion of genetically untested at-risk individuals.