Contributions
Abstract: P571
Type: Poster Sessions
Abstract Category: Therapy - Immunomodulation/Immunosuppression
Background: Biological processes involved in MS are largely shared across the age span. Despite a high relapse frequency in pediatric MS, very few drugs have been tested in that age group limiting the availability of safety and efficacy data. A randomized controlled phase 3 trial was completed in 2017 and 2 other phase 3 trials are ongoing. However, phase 3 trials for every newly approved DMT for adult MS are simply not feasible in children. Consideration should be given as to the best means to confirm efficacy and optimize safety for pediatric use.
Objective: To develop consensus recommendations for clinical trial designs that can deliver high quality data in pediatric MS patients.
Methods: The IPMSSG Steering Committee convened a meeting of experts in January 2018 to review the advances over the past six years in the understanding of pediatric-onset MS and issues faced in completed or ongoing clinical trials in that population.
Results: Challenges with recruitment, retention, study visits and choice of a control arm were identified that were specific to clinical trials in pediatric MS. The group considered how to improve study design and conduct based on specificities in this age group. Appropriate trial options for various categories of drug to be studied in pediatric MS were identified to address recruitment issues and avoid “ghost trials”. The group agreed that clinical trials in children with MS must consider pediatric-specific safety concerns both during the trial and in longer term open-label observation. IPMSSG Steering Committee recommendations will be presented regarding strategies for achieving these objectives, as well as endorsement of these recommendations by the IPMSSG membership.
Conclusions: These recommendations aim to ensure high quality evidence-based treatment for children and adolescents with MS to reduce reliance on off-label use, increase safety data and remove regulatory or insurance-based limitations in access to treatment.
Disclosure: Emmanuel 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.
Brenda Banwell has served as a central MRI reviewer for Novartis. Dr. Banwell serves as a non-remunerated advisor on clinical trial design for Novartis, Biogen-IDEC, Sanofi and Teva Neuroscience.
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 Maria Pia Sormani has received consulting fees from Biogen, TEVA, Merck, Novartis, Genzyme, Roche, Medday, GeNeuro, Actelion, Celgene
Maria Pia Amato has served on advisory boards for Biogen, Byer, Novartis, Merk, Teva, Sanofi- Genzyme, Roche and has received financial support for research activities from Biogen, Merk, Teva, Sanofi- Genzyme. She has received research grants from the Italian MS Society.
Rogier Hintzen has received honoraria for serving on advisory boards for Biogen Idec, Roche, Sanofi. He participated in trials with BiogenIdec, Merck-Serono, Roche, Genzyme and Novartis
Lauren Krupp has received research support, consulting fees, or royalties from the following entities: National Multiple Sclerosis Society, Department of Defense, Biogen, Eisai, IPSOS, Atara Biotherapeutics, ERT Inc, Pfizer, Everyday Health, Sanofi Aventis, RedHill Biopharma, Shire, Gerson Lehaman, Reata Pharma, Abbive, Amicus Therapeutics, Finkhar Health, Janssen Pharmaceuticals, Research Technology Kevin Rostasy has served on the advisory board for the clinical trial of Novartis and has received honoraria for invited talks from Merck.
Silvia Tenembaum serves as a non-remunerated editorial board member of Neurology: Neuroimmunology & Neuroinflammation. She has received speaker honoraria from Biogen-Idec Argentina, Merck Serono LATAM, Genzyme, Novartis, and Teva Neuroscience. 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.
Abstract: P571
Type: Poster Sessions
Abstract Category: Therapy - Immunomodulation/Immunosuppression
Background: Biological processes involved in MS are largely shared across the age span. Despite a high relapse frequency in pediatric MS, very few drugs have been tested in that age group limiting the availability of safety and efficacy data. A randomized controlled phase 3 trial was completed in 2017 and 2 other phase 3 trials are ongoing. However, phase 3 trials for every newly approved DMT for adult MS are simply not feasible in children. Consideration should be given as to the best means to confirm efficacy and optimize safety for pediatric use.
Objective: To develop consensus recommendations for clinical trial designs that can deliver high quality data in pediatric MS patients.
Methods: The IPMSSG Steering Committee convened a meeting of experts in January 2018 to review the advances over the past six years in the understanding of pediatric-onset MS and issues faced in completed or ongoing clinical trials in that population.
Results: Challenges with recruitment, retention, study visits and choice of a control arm were identified that were specific to clinical trials in pediatric MS. The group considered how to improve study design and conduct based on specificities in this age group. Appropriate trial options for various categories of drug to be studied in pediatric MS were identified to address recruitment issues and avoid “ghost trials”. The group agreed that clinical trials in children with MS must consider pediatric-specific safety concerns both during the trial and in longer term open-label observation. IPMSSG Steering Committee recommendations will be presented regarding strategies for achieving these objectives, as well as endorsement of these recommendations by the IPMSSG membership.
Conclusions: These recommendations aim to ensure high quality evidence-based treatment for children and adolescents with MS to reduce reliance on off-label use, increase safety data and remove regulatory or insurance-based limitations in access to treatment.
Disclosure: Emmanuel 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.
Brenda Banwell has served as a central MRI reviewer for Novartis. Dr. Banwell serves as a non-remunerated advisor on clinical trial design for Novartis, Biogen-IDEC, Sanofi and Teva Neuroscience.
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 Maria Pia Sormani has received consulting fees from Biogen, TEVA, Merck, Novartis, Genzyme, Roche, Medday, GeNeuro, Actelion, Celgene
Maria Pia Amato has served on advisory boards for Biogen, Byer, Novartis, Merk, Teva, Sanofi- Genzyme, Roche and has received financial support for research activities from Biogen, Merk, Teva, Sanofi- Genzyme. She has received research grants from the Italian MS Society.
Rogier Hintzen has received honoraria for serving on advisory boards for Biogen Idec, Roche, Sanofi. He participated in trials with BiogenIdec, Merck-Serono, Roche, Genzyme and Novartis
Lauren Krupp has received research support, consulting fees, or royalties from the following entities: National Multiple Sclerosis Society, Department of Defense, Biogen, Eisai, IPSOS, Atara Biotherapeutics, ERT Inc, Pfizer, Everyday Health, Sanofi Aventis, RedHill Biopharma, Shire, Gerson Lehaman, Reata Pharma, Abbive, Amicus Therapeutics, Finkhar Health, Janssen Pharmaceuticals, Research Technology Kevin Rostasy has served on the advisory board for the clinical trial of Novartis and has received honoraria for invited talks from Merck.
Silvia Tenembaum serves as a non-remunerated editorial board member of Neurology: Neuroimmunology & Neuroinflammation. She has received speaker honoraria from Biogen-Idec Argentina, Merck Serono LATAM, Genzyme, Novartis, and Teva Neuroscience. 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.