Longitudinal study of informed consent in innovative therapy research: experience and provisional recommendations from a multicenter trial of intracerebral grafting

PLoS One. 2015 May 26;10(5):e0128209. doi: 10.1371/journal.pone.0128209. eCollection 2015.

Abstract

Background: There is an urgent need to assess and improve the consent process in clinical trials of innovative therapies for neurodegenerative disorders.

Methods: 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. 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.

Results: The 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.

Conclusions: Cognitively 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.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Allografts
  • Belgium
  • Female
  • Follow-Up Studies
  • France
  • Humans
  • Huntington Disease / therapy*
  • Informed Consent*
  • Male
  • Middle Aged
  • Neural Stem Cells / transplantation*
  • Stem Cell Transplantation*
  • Surveys and Questionnaires*

Grants and funding

The MIG-HD trial is funded by two grants (AOM00139 and AOM 04021) from Programme Hospitalier de Recherche Clinique (Ministère de la Santé de France), and is sponsored by Assistance Publique - Hôpitaux de Paris (Département de la Recherche Clinique et du Développement) and by Association Française de lutte contre les Myopathies in Belgium. LCL was supported by an AP-HP/CEA interface contract. SS was supported by the NeuroStemCell Consortium (European Community's Seventh Framework Program grant agreement no. 222943). 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-10-IDEX-0001-02 PSL* grants. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.