Accepted for/Published in: JMIR Research Protocols
Date Submitted: Apr 24, 2020
Date Accepted: Jun 4, 2020
Date Submitted to PubMed: Jun 5, 2020
Study Protocol of AMASIA, a Non-Interventional Study to Gain First Insights on SPMS Patients Treated With Siponimod in Clinical Routine
ABSTRACT
Background:
A high proportion of patients with relapsing remitting multiple sclerosis (RRMS) convert to secondary progressive multiple sclerosis (SPMS) characterized by irreversibly progressing disability and cognitive decline. Siponimod (Mayzent®), a selective sphingosine-1-phosphate receptor modulator, has been recently approved by the EMA for the treatment of adult SPMS patients with active disease, as evidenced by relapses or magnetic resonance imaging features of ongoing inflammatory activity. Approval by the FDA covers a broader range of indications, comprising clinically isolated syndrome, RRMS, and active SPMS. However, treatment effects of siponimod have not been assessed in a structured setting in clinical routine so far.
Objective:
The objectives of AMASIA (ImpAct of Mayzent® (siponimod) on secondAry progressive multiple Sclerosis patients in a long-term non-Interventional study in GermAny), a prospective non-interventional study (NIS), are to assess long-term effectiveness and safety of siponimod in clinical routine, and to evaluate the impact of disease burden on quality of life and socioeconomic conditions. Here, we report the study design of AMASIA.
Methods:
Treatment effects of siponimod will be evaluated in 1500 SPMS patients during a 3-year observational phase. According to the genetic polymorphism of CYP2C9, the initial dose will be titrated to the maintenance dose of 1 mg (CYP2C9*1*3 and *2*3) or 2 mg (all other polymorphisms of CYP2C9 except *3*3 which is contraindicated) taken orally once daily. Primary endpoint is the 6-month confirmed disability progression, as assessed by a functional composite endpoint comprising the expanded disability status scale (EDSS) and the symbol digit modalities test (SDMT) to take appropriate account of cognitive changes and to increase sensitivity. Further measures including MS activity data, assessments of functional domains, questionnaires addressing the patient’s, physician’s, and relatives’ perspectives of disability progression, cognitive worsening, and quality of life as well as socioeconomic aspects will be documented by the MSDS3D system.
Results:
AMASIA is being conducted between February 2020 and February 2025 in up to 250 neurological centers in Germany.
Conclusions:
AMASIA will complement the pivotal phase-III-derived efficacy and safety profile of siponimod by real-world data and will further evaluate several individual treatment aspects such as quality of life and socioeconomic conditions of patients and care givers. It might help to establish siponimod as promising option for the treatment of SPMS patients in clinical routine.
Citation
Request queued. Please wait while the file is being generated. It may take some time.
Copyright
© The authors. All rights reserved. This is a privileged document currently under peer-review/community review (or an accepted/rejected manuscript). Authors have provided JMIR Publications with an exclusive license to publish this preprint on it's website for review and ahead-of-print citation purposes only. While the final peer-reviewed paper may be licensed under a cc-by license on publication, at this stage authors and publisher expressively prohibit redistribution of this draft paper other than for review purposes.