New study may help design future Huntington’s disease trials

25th April 2019 (Last Updated April 25th, 2019 13:16)

Researchers from the Perelman School of Medicine at the University of Pennsylvania (Penn) have conducted a new study focused on enabling better design of clinical trials for Huntington’s disease patients.

Performed in the form of a survey, the study involved patients at various stages of disease and examined their opinions and treatment objectives.

The researchers aimed to understand patients’ views on trial designs that involve non-standard therapies such as intravenous infusions, lumbar punctures or brain surgery.

Penn Neurology department senior genetic counsellor Tanya Bardakjian said: “In order to optimise feasibility, it is important to first understand how potential study participants feel about these new trial designs, with interventions that might involve a higher potential risk but also higher potential reward.”

Furthermore, the study was intended to analyse the responses of different individuals – a potential disease carrier, an asymptomatic genetic carrier and those with Huntington’s disease symptoms.

Out of the 87 respondents who completed the survey of hypothetical questions, 36 were diagnosed with the disease, 18 were carriers of pre-manifesting mutation and 33 were at-risk asymptomatic participants.

In addition to demographic, clinical and genetic background information, the questionnaire captured data on willingness to participate in certain types of trials and opinions about an investigator’s motivation.

While most of the respondents were positive towards participating in a trial, irrespective of design or therapy goals, asymptomatic genetic carriers were observed to be the most willing.

Across all groups, trials involving a placebo were viewed less favourably. Bardakjian said that an option to receive active treatment after the placebo period may address the negative views on a placebo group.

The results, which have been published in the Journal of Huntington’s Disease by the IOS Press, are expected to aid in designing future trials of gene therapies for Huntington’s disease and other genetic disorders.