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J Huntingtons Dis. 2019;8(1):79-85. doi: 10.3233/JHD-180328.

Attitudes of Potential Participants Towards Molecular Therapy Trials in Huntington's Disease.

Author information

1
Department of Neurology, Hospital of the University of Pennsylvania, Philadelphia, PA, USA.
2
Raymond G. Perelman Center for Cellular & Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.

Abstract

BACKGROUND:

Advances in molecular therapeutic approaches in the last decade are translating into the design of non-traditional clinical trials. In order to improve their feasibility, it is important to understand the attitudes of potential participants towards these trials, their motivations to get involved and acceptance of risks.

OBJECTIVE:

We aimed to better understand the willingness of potential participants to participate in different molecular therapy trials for Huntington's disease (HD) based on their clinical and genetic status, trial design and goals of the treatment.

METHODS:

An anonymous survey was distributed through the Huntington's Disease Society of America (HDSA) on-line portal/website. Various hypothetical scenarios were presented followed by a survey consistent of Likert scale responses ascertaining willingness to participate, collecting demographic, clinical and genetic information.

RESULTS:

There were a total of 87 responses, including patients diagnosed with HD, pre-manifesting mutation carriers and asymptomatic participants at risk. The majority of participants indicated they were very likely or likely to participate in clinical trials independent of study design or goals of the therapy, with a more favorable view in premanifesting mutation carriers. However, more invasive procedures and trials including placebo were less favorably viewed across all diagnostic groups.

CONCLUSIONS:

In summary, most individuals in the HD community would consider participation in novel molecular therapy trials, but study design and goals could impact patient recruitment. This data can be used to inform the recruitment and consent process into clinical trials and to address common concerns by potential participants.

KEYWORDS:

Huntington’s disease; clinical trial; gene therapy

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