Dataset Information

International Pediatric MS Study Group Clinical Trials Summit: meeting report.

ABSTRACT:

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.

SUBMITTER: Chitnis T

PROVIDER: S-EPMC3662305 | biostudies-literature | 2013 Mar

REPOSITORIES: biostudies-literature

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Publications

International Pediatric MS Study Group Clinical Trials Summit: meeting report.

Chitnis Tanuja T Tardieu Marc M Amato Maria Pia MP Banwell Brenda B Bar-Or Amit A Ghezzi Angelo A Kornberg Andrew A Krupp Lauren B LB Pohl Daniela D Rostasy Kevin K Tenembaum Silvia S Waubant Emmanuelle E Wassmer Evangeline E

Neurology 20130301 12

<h4>Objective</h4>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 ...[more]

PMID: 23509048

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Dataset Information

International Pediatric MS Study Group Clinical Trials Summit: meeting report.

Objective

Methods

Results

Conclusion

Publications

International Pediatric MS Study Group Clinical Trials Summit: meeting report.

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