International Pediatric MS Study Group Clinical Trials Summit Meeting report

被引：44

作者：

Chitnis, Tanuja ^{[1
]}

Tardieu, Marc ^{[2
]}

Amato, Maria Pia ^{[3
]}

Banwell, Brenda ^{[4
]}

Bar-Or, Amit ^{[5
]}

Ghezzi, Angelo ^{[6
]}

Kornberg, Andrew ^{[7
]}

Krupp, Lauren B. ^{[8
]}

Pohl, Daniela ^{[9
]}

Rostasy, Kevin ^{[10
]}

Tenembaum, Silvia ^{[11
]}

Waubant, Emmanuelle ^{[12
]}

Wassmer, Evangeline ^{[13
]}

机构：

[1] Massachusetts Gen Hosp, Partners Pediat Multiple Sclerosis Ctr, Boston, MA 02114 USA

[2] Hop Univ Paris Sud, Assistance Publ Hop Paris, Paris, France

[3] Univ Florence, Dept Neurol, Florence, Italy

[4] Univ Toronto, Hosp Sick Children, Toronto, ON M5G 1X8, Canada

[5] McGill Univ, Montreal Neurol Inst & Hosp, Montreal, PQ, Canada

[6] Hosp Gallarate, Multiple Sclerosis Study Ctr, Gallarate, Italy

[7] Royal Childrens Hosp, Parkville, Vic 3052, Australia

[8] Lourie Ctr Pediat MS, Stony Brook, NY USA

[9] Univ Ottawa, Childrens Hosp Eastern Ontario, Ottawa, ON, Canada

[10] Med Univ Innsbruck, Dept Pediat 4, Div Pediat Neurol & Inborn Errors Metab, A-6020 Innsbruck, Austria

[11] Natl Pediat Hosp, Buenos Aires, DF, Argentina

[12] Univ Calif San Francisco, MS Ctr, San Francisco, CA 94143 USA

[13] Birmingham Childrens Hosp, Birmingham, W Midlands, England

来源：

NEUROLOGY | 2013年 / 80卷 / 12期

关键词：

ONSET MULTIPLE-SCLEROSIS; DEMYELINATING SYNDROMES; PSYCHOSOCIAL FEATURES; CHILDHOOD-ONSET; JUVENILE MS; CHILDREN; DISEASE; LESIONS; COHORT; ADULTS;

D O I：

10.1212/WNL.0b013e318288694e

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Objective: Pediatric studies for new biological agents are mandated by recent legislation, necessitating careful thought to evaluation of emerging multiple sclerosis (MS) therapies in children with MS. Challenges include a small patient population, the lack of prior randomized clinical trials, and ethical concerns. The goal of this meeting was to assess areas of consensus regarding clinical trial design and outcome measures among academic experts involved in pediatric MS care and research. Methods: The Steering Committee of the International Pediatric MS Study Group identified key focus areas for discussion. A total of 69 meeting attendees were assembled, including 35 academic experts. Regulatory and pharmaceutical representatives also attended, and provided input, which informed academic expert consensus decisions. Results: The academic experts agreed that clinical trials were necessary in pediatric MS to obtain pharmacokinetic, safety and efficacy data, and regulatory approval allowing for greater medication access. The academic experts agreed that relapse was an appropriate primary outcome measure for phase III pediatric trials. An international standardized cognitive battery was identified. The pros and cons of various trial designs were discussed. Guidelines surrounding MRI studies, pharmacokinetics, pharmacodynamics, and registries were developed. The academic experts agreed that given the limited subject pool, a stepwise approach to the launch of clinical trials for the most promising medications is necessary in order to ensure study completion. Alternative approaches could result in unethical exposure of patients to trial conditions without gaining knowledge. Conclusion: Consensus points for conduct of clinical trials in the rare disease pediatric MS were identified amongst a panel of academic experts, informed by regulatory and industry stakeholders. Neurology (R) 2013; 80:1161-1168

引用

页码：1161 / 1168

页数：8

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