Abstract
BackgroundThere is an urgent need to assess and improve the consent process in clinical trials of innovative therapies for neurodegenerative disorders.MethodsWe performed a longitudinal study of the consent of Huntington’s disease patients during the Multicenter Fetal Cell Intracerebral Grafting Trial in Huntington’s Disease (MIG-HD) in France and Belgium. Patients and their proxies completed a consent questionnaire at inclusion, before signing the consent form and after one year of follow-up, before randomization and transplantation. The questionnaire explored understanding of the protocol, satisfaction with the information delivered, reasons for participating in the trial and expectations regarding the transplant. Forty-six Huntington’s disease patients and 27 proxies completed the questionnaire at inclusion, and 27 Huntington’s disease patients and 16 proxies one year later.ResultsThe comprehension score was high and similar for Huntington’s disease patients and proxies at inclusion (72.6% vs 77.8%; P > 0.1) but only decreased in HD patients after one year. The information satisfaction score was high (73.5% vs 66.5%; P > 0.1) and correlated with understanding in both patients and proxies. The motivation and expectation profiles were similar in patients and proxies and remained unchanged after one year.ConclusionsCognitively impaired patients with Huntington’s disease were capable of consenting to participation in this trial. This consent procedure has presumably strengthened their understanding and should be proposed before signing the consent form in future gene or cell therapy trials for neurodegenerative disorders. Because of the potential cognitive decline, proxies should be designated as provisional surrogate decision-makers, even in competent patients.
Highlights
Informed consent is a prerequisite for research on humans worldwide, but it is challenging to obtain meaningful consent in studies of highly innovative therapies, such as intracerebral cell grafting or gene therapy for neurodegenerative diseases
Therapeutic misconception—a failure to understand that the defining purpose of clinical research is to produce generalizable knowledge [6]—may be frequent in the setting of cell therapy [7]
We prospectively investigated whether the Huntington’s disease (HD) patients were able to provide valid consent and the role of proxies as potential surrogate decisionmakers
Summary
We performed a longitudinal study of the consent of Huntington’s disease patients during the Multicenter Fetal Cell Intracerebral Grafting Trial in Huntington’s Disease (MIG-HD) in France and Belgium. Patients and their proxies completed a consent questionnaire at inclusion, before signing the consent form and after one year of follow-up, before randomization and transplantation. Additional support was provided through the NeurATRIS project (European Advanced Translational Research Infrastructure in Neurosciences) and from Agence Nationale pour la Recherche ANR-10-LABX-0087 IEC and ANR-10IDEX-0001-02 PSLÃ grants.
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