Contributions
Abstract: P290
Type: Poster
Abstract Category: Clinical aspects of MS - Paediatric MS
Background: Approximately 3‒5% of patients with multiple sclerosis (MS) experience disease onset before18 years of age with 2‒3 times more frequent relapses than adults. PARADIGMS is the first global, controlled study investigating the efficacy and safety of fingolimod up to 0.5 mg vs interferon (IFN) β-1a in paediatric patients.
OBJECTIVE : To present baseline characteristics of the first 190 patients randomised in the PARADIGMS study.
Design and methods: PARADIGMS is an ongoing 24-month, double-blind, double-dummy, randomised, active-controlled, parallel-group, multicentre global study conducted in patients with MS aged between 10 to < 18 years. Patients were randomised to receive either oral fingolimod once-daily (dose adjusted for body weight) or intramuscular IFN β-1a 30 µg once weekly. Eligibility criteria included: MS diagnosis as defined by the revised consensus definition for paediatric MS consistent with 2010 McDonald criteria, at least 1 relapse during the previous year or 2 relapses in the previous two years or 1 or more gadolinium-enhancing (Gd+) lesions in the previous 6 months prior to enrolment. Expanded Disability Status Scale (EDSS) score of 0 to 5.5. Here we present the baseline demographic, disease and clinical characteristics.
Results: Baseline data are reported here for the first 190 randomised patients. The mean±SD age was 15.3±1.82 years, with more patients in the age group of >14 to ≤16 years (41%), followed by >16 years (31%) and 10% of patients were ≤12 years. The majority of patients were female (63%) and Caucasian (88%). Only 6 (3%) patients were pre-pubertal (Tanner Stage less than 2). The mean±SD duration of MS since first symptom was 2.1±1.92 years; mean number of relapses in the last 12 and 12‒24 months before screening was 1.5 and 0.9 respectively. Median EDSS at baseline was 1.5 (range 0.0-5.5). At baseline, the mean±SD number of Gd+ lesions was 3.1±6.56 with 49% of patients free from Gd+ lesions.
Conclusions: Paediatric MS patients enrolled in the PARADIGMS study were representative of known paediatric MS cohorts, with a high frequency of relapses and Gd+ lesions early in their course and had a preponderance of post-pubertal patients. The PARADIGMS study has successfully met the overall recruitment target. However recruitment remains open to enrol additional eligible pre-pubertal patients; updated numbers will be presented at the meeting.
Disclosure:
Funding source: This study is supported by Novartis Pharma AG, Basel, Switzerland. The following authors have received compensation for serving as consultants or speakers, or they or the institutions they work for have received research support from the companies indicated:
Tanuja Chitnis received personal compensation for advisory boards/consulting for Hoffman-La Roche, Biogen and Novartis Pharmaceuticals and financial support for research activities from Biogen, Merck Serono and Novartis Pharmaceuticals.
Douglas L Arnold has received honoraria from Acorda, Biogen Idec, Genentech, Genzyme, Novartis, Hoffman-La Roche and Sanofi-Aventis, has received research support from Novartis and Biogen, and has an equity interest in NeuroRx Research, which performed the MRI analysis for the trial.
Brenda Banwell has served as an unpaid consultant to Biogen-Idec, Novartis, Teva Neuroscience, and Merck-Serono; as a remunerated central MRI reviewer for the present trial, and she is Chief Editor for MS and Related Disorders and funded by Canadian MS scientific research foundation (CMSRF), Canadian Multiple Sclerosis Society (CMSS), National Multiple Sclerosis Society (NMSS), and Canadian Institutes of Health Research (CIHR).
Wolfgang Brück has received honoraria for lectures by Bayer Vital, Biogen, Merck Serono, Teva Pharma, Genzyme, Sanofi-Aventis and Novartis and is a member of scientific advisory boards for Teva Pharma, Biogen, Novartis and Genzyme. He received funding for research projects by Teva Pharma, Biogen, Novartis and Genzyme. Dr Brück serves on the editorial boards of Neuropathology and Applied Neurobiology, Multiple Sclerosis International and Therapeutic Advances in Neurological Disorders.
Angelo Ghezzi received honoraria for speaking from Almirall, Biogen Idec, Merck Serono, Novartis, Genzyme and Sanofi-Aventis; and for consultancy from Merck Serono, Biogen Idec, Teva and Novartis Pharmaceuticals.
Gavin Giovannoni has received fees for participation in advisory board for AbbVie Biotherapeutics Inc., Biogen, Canbex, Ironwood, Novartis, Merck, Merck Serono, Roche, SanofiGenzyme, Synthon, Teva, and Vertex; speaker fees from AbbVie Biotherapeutics Inc., Biogen, Bayer HealthCare, Genzyme, Merck Serono, SanofiAventis, and Teva; coeditor in chief of Multiple Sclerosis and Related Disorders; research support unrelated to study from Biogen, Genzyme, Ironwood, Merck Serono, and Novartis.
Benjamin Greenberg has received compensation for consulting services from EMD Serono and Novartis. He has received grant support from Chugai, Biogen, Acorda, Medimmune, PCORI, NIH and Guthy Jackson Charitable Foundation.
Lauren Krupp has received personal compensation for activities as a speaker, consultant and/or participant on an advisory board from Biogen Idec, Novartis Pharmaceuticals, Teva Neurosciences, and Multicell; royalty or license fees from ER Squibb & Sons, Avenir, Johnson & Johnson, and Osmotica; grant support from the National Multiple Sclerosis Society, National Institutes of Health, and the Department of Defense; and research support from Novartis, Biogen Idec, Celgene Corporation, and Genentech. She has also received support from the Lourie Foundation, Slomo and Cindy Silvian Foundation, and the Multiple Sclerosis Foundation.
Kevin Rostasy has served as consultant for PARADIGMS study. Received sponsoring for lecture and symposia from Merck Serono.
Marc Tardieu is a member of scientific boards for Novartis, Sanofi and Uniqure.
Emmanuelle Waubant is funded by the NIH, NMSS and Race to Erase MS. She volunteers on an advisory board for a Novartis trial. She is site PI for clinical trials with Roche and Novartis.
Jerry Wolinsky in the last 3 years has received compensation for service on steering committees or data monitoring boards for F. Hoffmann-La Roche Ltd., Medday Pharmaceuticals, Novartis, Sanofi Genzyme and Teva Pharmaceuticals; consultant fees from AbbVie, Actelion, Alkermes, EMD Serono, Forward Pharma, Genentech, Inc., F. Hoffmann-La Roche Ltd., Novartis, Sanofi Genzyme, Takeda, Teva, and XenoPort; research support from, Sanofi Genzyme, the NIH and the NMSS through the University of Texas Health Science Center at Houston (UTHSCH) and royalties for monoclonal antibodies out-licensed to Chemicon International through UTHSCH.
Amit Bar-Or has participated as a speaker at meetings sponsored by, received consulting fees and/or received grant support from: Amplimmune, Bayhill Therapeutics, Berlex/Bayer, Biogen Idec, Diogenix, Eli-Lilly, Genentech, GlaxoSmithKline, Guthy-Jackson/GGF, Merck/EMD Serono, Medimmune, Mitsubishi Pharma, Novartis, Ono Pharma, Receptos, Roche, Sanofi-Genzyme, Teva Neuroscience, Wyeth.
Jutta Gärtner has received honoraria and consultancy fees from Bayer Vital, Biogen, Merck Serono, Teva, and Novartis and has received research grant support from Novartis and Biogen.
Tracy Stites and Martin Merschhemke are employees of Novartis.
Abstract: P290
Type: Poster
Abstract Category: Clinical aspects of MS - Paediatric MS
Background: Approximately 3‒5% of patients with multiple sclerosis (MS) experience disease onset before18 years of age with 2‒3 times more frequent relapses than adults. PARADIGMS is the first global, controlled study investigating the efficacy and safety of fingolimod up to 0.5 mg vs interferon (IFN) β-1a in paediatric patients.
OBJECTIVE : To present baseline characteristics of the first 190 patients randomised in the PARADIGMS study.
Design and methods: PARADIGMS is an ongoing 24-month, double-blind, double-dummy, randomised, active-controlled, parallel-group, multicentre global study conducted in patients with MS aged between 10 to < 18 years. Patients were randomised to receive either oral fingolimod once-daily (dose adjusted for body weight) or intramuscular IFN β-1a 30 µg once weekly. Eligibility criteria included: MS diagnosis as defined by the revised consensus definition for paediatric MS consistent with 2010 McDonald criteria, at least 1 relapse during the previous year or 2 relapses in the previous two years or 1 or more gadolinium-enhancing (Gd+) lesions in the previous 6 months prior to enrolment. Expanded Disability Status Scale (EDSS) score of 0 to 5.5. Here we present the baseline demographic, disease and clinical characteristics.
Results: Baseline data are reported here for the first 190 randomised patients. The mean±SD age was 15.3±1.82 years, with more patients in the age group of >14 to ≤16 years (41%), followed by >16 years (31%) and 10% of patients were ≤12 years. The majority of patients were female (63%) and Caucasian (88%). Only 6 (3%) patients were pre-pubertal (Tanner Stage less than 2). The mean±SD duration of MS since first symptom was 2.1±1.92 years; mean number of relapses in the last 12 and 12‒24 months before screening was 1.5 and 0.9 respectively. Median EDSS at baseline was 1.5 (range 0.0-5.5). At baseline, the mean±SD number of Gd+ lesions was 3.1±6.56 with 49% of patients free from Gd+ lesions.
Conclusions: Paediatric MS patients enrolled in the PARADIGMS study were representative of known paediatric MS cohorts, with a high frequency of relapses and Gd+ lesions early in their course and had a preponderance of post-pubertal patients. The PARADIGMS study has successfully met the overall recruitment target. However recruitment remains open to enrol additional eligible pre-pubertal patients; updated numbers will be presented at the meeting.
Disclosure:
Funding source: This study is supported by Novartis Pharma AG, Basel, Switzerland. The following authors have received compensation for serving as consultants or speakers, or they or the institutions they work for have received research support from the companies indicated:
Tanuja Chitnis received personal compensation for advisory boards/consulting for Hoffman-La Roche, Biogen and Novartis Pharmaceuticals and financial support for research activities from Biogen, Merck Serono and Novartis Pharmaceuticals.
Douglas L Arnold has received honoraria from Acorda, Biogen Idec, Genentech, Genzyme, Novartis, Hoffman-La Roche and Sanofi-Aventis, has received research support from Novartis and Biogen, and has an equity interest in NeuroRx Research, which performed the MRI analysis for the trial.
Brenda Banwell has served as an unpaid consultant to Biogen-Idec, Novartis, Teva Neuroscience, and Merck-Serono; as a remunerated central MRI reviewer for the present trial, and she is Chief Editor for MS and Related Disorders and funded by Canadian MS scientific research foundation (CMSRF), Canadian Multiple Sclerosis Society (CMSS), National Multiple Sclerosis Society (NMSS), and Canadian Institutes of Health Research (CIHR).
Wolfgang Brück has received honoraria for lectures by Bayer Vital, Biogen, Merck Serono, Teva Pharma, Genzyme, Sanofi-Aventis and Novartis and is a member of scientific advisory boards for Teva Pharma, Biogen, Novartis and Genzyme. He received funding for research projects by Teva Pharma, Biogen, Novartis and Genzyme. Dr Brück serves on the editorial boards of Neuropathology and Applied Neurobiology, Multiple Sclerosis International and Therapeutic Advances in Neurological Disorders.
Angelo Ghezzi received honoraria for speaking from Almirall, Biogen Idec, Merck Serono, Novartis, Genzyme and Sanofi-Aventis; and for consultancy from Merck Serono, Biogen Idec, Teva and Novartis Pharmaceuticals.
Gavin Giovannoni has received fees for participation in advisory board for AbbVie Biotherapeutics Inc., Biogen, Canbex, Ironwood, Novartis, Merck, Merck Serono, Roche, SanofiGenzyme, Synthon, Teva, and Vertex; speaker fees from AbbVie Biotherapeutics Inc., Biogen, Bayer HealthCare, Genzyme, Merck Serono, SanofiAventis, and Teva; coeditor in chief of Multiple Sclerosis and Related Disorders; research support unrelated to study from Biogen, Genzyme, Ironwood, Merck Serono, and Novartis.
Benjamin Greenberg has received compensation for consulting services from EMD Serono and Novartis. He has received grant support from Chugai, Biogen, Acorda, Medimmune, PCORI, NIH and Guthy Jackson Charitable Foundation.
Lauren Krupp has received personal compensation for activities as a speaker, consultant and/or participant on an advisory board from Biogen Idec, Novartis Pharmaceuticals, Teva Neurosciences, and Multicell; royalty or license fees from ER Squibb & Sons, Avenir, Johnson & Johnson, and Osmotica; grant support from the National Multiple Sclerosis Society, National Institutes of Health, and the Department of Defense; and research support from Novartis, Biogen Idec, Celgene Corporation, and Genentech. She has also received support from the Lourie Foundation, Slomo and Cindy Silvian Foundation, and the Multiple Sclerosis Foundation.
Kevin Rostasy has served as consultant for PARADIGMS study. Received sponsoring for lecture and symposia from Merck Serono.
Marc Tardieu is a member of scientific boards for Novartis, Sanofi and Uniqure.
Emmanuelle Waubant is funded by the NIH, NMSS and Race to Erase MS. She volunteers on an advisory board for a Novartis trial. She is site PI for clinical trials with Roche and Novartis.
Jerry Wolinsky in the last 3 years has received compensation for service on steering committees or data monitoring boards for F. Hoffmann-La Roche Ltd., Medday Pharmaceuticals, Novartis, Sanofi Genzyme and Teva Pharmaceuticals; consultant fees from AbbVie, Actelion, Alkermes, EMD Serono, Forward Pharma, Genentech, Inc., F. Hoffmann-La Roche Ltd., Novartis, Sanofi Genzyme, Takeda, Teva, and XenoPort; research support from, Sanofi Genzyme, the NIH and the NMSS through the University of Texas Health Science Center at Houston (UTHSCH) and royalties for monoclonal antibodies out-licensed to Chemicon International through UTHSCH.
Amit Bar-Or has participated as a speaker at meetings sponsored by, received consulting fees and/or received grant support from: Amplimmune, Bayhill Therapeutics, Berlex/Bayer, Biogen Idec, Diogenix, Eli-Lilly, Genentech, GlaxoSmithKline, Guthy-Jackson/GGF, Merck/EMD Serono, Medimmune, Mitsubishi Pharma, Novartis, Ono Pharma, Receptos, Roche, Sanofi-Genzyme, Teva Neuroscience, Wyeth.
Jutta Gärtner has received honoraria and consultancy fees from Bayer Vital, Biogen, Merck Serono, Teva, and Novartis and has received research grant support from Novartis and Biogen.
Tracy Stites and Martin Merschhemke are employees of Novartis.