Contributions
Abstract: P349
Type: Poster Sessions
Abstract Category: Clinical aspects of MS - Paediatric MS
Introduction and objective: For the first time in the past 5 years, pediatric multiple sclerosis (MS) trials have been launched, with some fully enrolled or completed. The experience of investigators with these recent trials has provided new insights on the feasibility and challenges of studies in pediatric MS, which are important to understand for future trials.
Method: We conducted a survey of the International Pediatric Multiple Sclerosis Study Group membership regarding barriers to pediatric MS clinical trials, using a web-based tool.
Results: 58 care providers from 18 countries answered the survey of which 38 had participated in a clinical trial. Top challenges for providers included the time to set up the trial and obtain local ethics approval, the frequency of study visits and difficulty balancing such effort with clinical time, and limited access to part-time clinical trial coordinator support. The frequency of study visits proved a barrier to patient recruitment and retention. Ethical concerns, raised by both providers and families, included the use of placebo, or comparator trials using a drug shown to be inferior to study drug in adult MS trials. Providers recommended strategies to reduce the number of in-person visits, shorten the time of research visits, enable laboratory sampling at sites close to home, and to limit the number MRI scans required.
Conclusions: This study highlights important barriers to success in pediatric MS clinical trials, and provides suggestions for study design that would be more practical for families and clinical trial staff.
Disclosure: Evangeline Wassmer has served as a consultant for Novartis and Biogen, She is an investigator in trials with Biogen Idec, Sanofi and Novartis. Her MS research projects have been funded by the UK MS Society, Action Medical Research and Birmingham Children's Hospital Research Foundation.
Brenda Banwell has served as a consultant for Novartis. She is a non-remunerated advisor for clinical trial design to Novartis, Biogen-IDEC, Sanofi, and Teva Neuroscience. She is funded by the Canadian Multiple Sclerosis Research Foundation, NMSS, and PCORI.
Tanuja Chitnis has served on the advisory boards for clinical trials for Novartis and Sanofi-Genzyme. She has received compensation for advisory/consulting boards for Biogen, Novartis and Sanofi-Genzyme. She has received financial support for research activities from Merck-Serono and Verily.
Emmanuelle Waubant has not received any pharmaceutical company honorarium. She is site PI for a Novartis and Roche trial and has volunteered on an advisory board for a Novartis trial. She is a non-remunerated advisor for clinical trial design to Novartis, Biogen-IDEC, Sanofi, Genentech, Serono and Celgene. She has funding from the NMSS, PCORI and the Race to Erase MS. She is the section editor for Annals of Clinical and Translational Neurology, and co-Chief editor for MSARD.
Kumaran Deiva is an investigator in trials with Novartis, Sanofi and Biogen and received consultancy fees from Biogen, Novartis, Sanofi.
Abstract: P349
Type: Poster Sessions
Abstract Category: Clinical aspects of MS - Paediatric MS
Introduction and objective: For the first time in the past 5 years, pediatric multiple sclerosis (MS) trials have been launched, with some fully enrolled or completed. The experience of investigators with these recent trials has provided new insights on the feasibility and challenges of studies in pediatric MS, which are important to understand for future trials.
Method: We conducted a survey of the International Pediatric Multiple Sclerosis Study Group membership regarding barriers to pediatric MS clinical trials, using a web-based tool.
Results: 58 care providers from 18 countries answered the survey of which 38 had participated in a clinical trial. Top challenges for providers included the time to set up the trial and obtain local ethics approval, the frequency of study visits and difficulty balancing such effort with clinical time, and limited access to part-time clinical trial coordinator support. The frequency of study visits proved a barrier to patient recruitment and retention. Ethical concerns, raised by both providers and families, included the use of placebo, or comparator trials using a drug shown to be inferior to study drug in adult MS trials. Providers recommended strategies to reduce the number of in-person visits, shorten the time of research visits, enable laboratory sampling at sites close to home, and to limit the number MRI scans required.
Conclusions: This study highlights important barriers to success in pediatric MS clinical trials, and provides suggestions for study design that would be more practical for families and clinical trial staff.
Disclosure: Evangeline Wassmer has served as a consultant for Novartis and Biogen, She is an investigator in trials with Biogen Idec, Sanofi and Novartis. Her MS research projects have been funded by the UK MS Society, Action Medical Research and Birmingham Children's Hospital Research Foundation.
Brenda Banwell has served as a consultant for Novartis. She is a non-remunerated advisor for clinical trial design to Novartis, Biogen-IDEC, Sanofi, and Teva Neuroscience. She is funded by the Canadian Multiple Sclerosis Research Foundation, NMSS, and PCORI.
Tanuja Chitnis has served on the advisory boards for clinical trials for Novartis and Sanofi-Genzyme. She has received compensation for advisory/consulting boards for Biogen, Novartis and Sanofi-Genzyme. She has received financial support for research activities from Merck-Serono and Verily.
Emmanuelle Waubant has not received any pharmaceutical company honorarium. She is site PI for a Novartis and Roche trial and has volunteered on an advisory board for a Novartis trial. She is a non-remunerated advisor for clinical trial design to Novartis, Biogen-IDEC, Sanofi, Genentech, Serono and Celgene. She has funding from the NMSS, PCORI and the Race to Erase MS. She is the section editor for Annals of Clinical and Translational Neurology, and co-Chief editor for MSARD.
Kumaran Deiva is an investigator in trials with Novartis, Sanofi and Biogen and received consultancy fees from Biogen, Novartis, Sanofi.