Design of a non-interventional post-marketing study to assess the long-term safety and effectiveness of ocrelizumab in German real world multiple sclerosis cohorts – the CONFIDENCE study protocol

Autor: David Wormser, Jost Leemhuis, Stefanie Hieke-Schulz, Vera Zingler, Tjalf Ziemssen, Petra Dirks, Heike Berthold
Jazyk: angličtina
Rok vydání: 2020
Předmět:
Long-term safety and effectiveness
medicine.medical_specialty
Real world data
Antibodies
Monoclonal
Humanized
Non-interventional study
lcsh:RC346-429
Study Protocol
03 medical and health sciences
Multiple Sclerosis
Relapsing-Remitting
0302 clinical medicine
Primary progressive multiple sclerosis
Germany
Product Surveillance
Postmarketing
medicine
Clinical endpoint
Humans
Prospective Studies
030212 general & internal medicine
Ocrelizumab
Adverse effect
lcsh:Neurology. Diseases of the nervous system
Protocol (science)
Disease modifying therapy
business.industry
Multiple sclerosis
Incidence (epidemiology)
MSDS3
General Medicine
Multiple Sclerosis
Chronic Progressive
medicine.disease
3. Good health
Clinical trial
Research Design
Emergency medicine
Relapsing multiple sclerosis
Neurology (clinical)
Neurosurgery
business
030217 neurology & neurosurgery
medicine.drug
Zdroj: BMC Neurology, Vol 20, Iss 1, Pp 1-9 (2020)
BMC Neurology
ISSN: 1471-2377
Popis: Background Multiple sclerosis (MS) is a chronic disease that requires lifelong treatment. A highly effective drug not only for relapsing but also for progressive forms of MS with a favorable safety profile is needed to further improve overall patient outcomes. Ocrelizumab, a recombinant humanized monoclonal antibody that selectively targets CD20-expressing B-cells, is the first drug indicated for the treatment of adult patients with relapsing forms of MS (RMS) and primary progressive MS (PPMS). Its safety and effectiveness profile has yet to be studied in a large, real-world setting. CONFIDENCE aims to further characterize the safety profile of ocrelizumab in routine clinical practice. In addition, real-world effectiveness data will be collected to complement the efficacy data documented in the pivotal clinical trials. Methods CONFIDENCE is a non-interventional, prospective, multicenter, long-term study collecting primary data from 3000 RMS and PPMS patients newly treated with ocrelizumab and 1500 patients newly treated with other selected MS disease-modifying therapies (DMTs). Treatment must be in accordance with the local label and follow routine practice. Data will be collected at approximately 250 neurological centers and practices across Germany. The recruitment period of 30 months started in April 2018. The observation period per patient is planned 7.5 to 10 years, depending on the date of inclusion, regardless of whether patients discontinue treatment. Visits follow routine practice and will be documented approximately every 6 months. The primary endpoint is the incidence and type of uncommon adverse events and death. Statistical analyses will be mainly descriptive and exploratory. Discussion CONFIDENCE is a large, non-interventional, post-authorization safety study that assesses long-term safety and effectiveness of ocrelizumab and other DMTs in a real-world setting. Data collected in CONFIDENCE will also be integrated into studies that have been developed to fulfil international regulatory requirements.
Databáze: OpenAIRE
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