Eligible Huntington’s disease patients would likely participate in a gene therapy trial regardless of the study design, but they are more inclined toward those with less invasive interventions and without a placebo group, a questionnaire-based study suggests.

The results may be useful to design more patient-centred clinical trials assessing the effects of gene therapy in Huntington’s patients.

The study, “Attitudes of Potential Participants Towards Molecular Therapy Trials in Huntington’s Disease,” was published in the Journal of Huntington’s Disease.

While a new therapy might face obstacles to its development during clinical trials, a significant proportion of studies fail because of the complexity of patient recruitment and lack of patient participation.

In order to optimize feasibility [of clinical trials], 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
Tanya M. Bardakjian, the study’s first author and a senior genetic counselor in the Department of Neurology at Perelman School of Medicine at the University of Pennsylvania, said in a press release.

Participants who knew they carried Huntington’s mutation and would eventually develop the disease were significantly more positive toward participation in clinical trials, except those with higher risk or lower benefit.

Despite the study’s limitations, such as a small sample size with highly educated participants, researchers say their findings likely reflect the global opinion of Huntington’s patients.

We believe that a patient-centered trial design, through the inclusion of appropriate education and communication to ensure participants and advocates are informed and engaged, is likely to have a positive impact on recruitment
Tanya M. Bardakjian


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