A Study to Assess the Effectiveness and Safety of Mayzent in Chinese Patients With Relapsing Forms of Multiple Sclerosis

A Real-World, Retrospective, Multicenter Study to Assess the Effectiveness and Safety of Mayzent® (Siponimod) in Chinese Patients With Relapsing Forms of Multiple Sclerosis

This was a multicenter, non-interventional, retrospective study aiming to evaluate the real-world effectiveness and safety of siponimod treatment in Chinese patients with relapsing forms of multiple sclerosis (RMS). The data were collected retrospectively through medical records review and abstraction conducted at a single time point per patient by the investigator's site staff or a designate (at the discretion of the site, if allowed by local regulations). There was no prospective patient follow-up for this study. Obtaining informed consent was based on local regulations. Where permissible, waivers could be applied to the Institutional Review Board (IRB) or Independent Ethics Committee (IEC) as appropriate, based on the retrospective collection of non-personally identifiable data, if acceptable per local regulations.

The target patient population included adult patients diagnosed with RMS (including clinically isolated syndrome (CIS), relapsing-remitting multiple sclerosis (RRMS), or active secondary progressive multiple sclerosis (SPMS)), and who received at least 3-months of treatment with siponimod after the index date. The index date is the date of siponimod initiation, defined as the date of first prescription record of siponimod in the patient's medical records with RMS diagnosis. Effectiveness data (i.e., clinical relapses, magnetic resonance imaging (MRI) activity) were collected from the index date, through the end of the observation period. The observation period was from the index date to the date of initiation of medical records abstraction at site, or patient withdrawal of consent, loss of follow-up, or death, whichever occurred first. Among patients who permanently discontinued siponimod during the observation period, safety data were collected up to 30 days after the last dose of siponimod.

Study Overview

• Status: Completed
• Conditions: Clinically Isolated Syndrome; Relapsing Multiple Sclerosis; Relapsing Remitting Multiple Sclerosis; Secondary Progressive Multiple Sclerosis

• Study Type: Observational
• Enrollment (Actual): 113

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • East Hanover, New Jersey, United States, 07936
      • Novartis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

Description

Inclusion criteria:

• Written informed consent if requested by local regulation.
• Patients had a diagnosis of RMS (including CIS, RRMS, or active SPMS).
• Chinese patients were 18 years or older at the index date.
• Patients had a minimum 3-month persistence on siponimod after the index date.
• Patients had at least one documented clinical visit in the observation period after the initiation of siponimod treatment.

Exclusion criteria:

• Patients previously treated with siponimod (including participation in clinical trials) before the index date.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Siponimod cohort; Patients who were at least 18 years old diagnosed with clinically isolated syndrome (CIS), relapsing-remitting multiple sclerosis (RRMS), or active secondary progressive multiple sclerosis and had received at least 3 months of siponimod treatment after the index date.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Relapse Rate (ARR)	The ARR was calculated using the total number of relapses (as documented in the patient's medical records) after index date divided by the total person days of follow-up after index date multiplied by 365.25.	Up to approximately 32 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Patients Relapse-free at 12 Months after Index Date		12 months
Proportion of Patients Free of Magnetic Resonance Imaging (MRI) Activity at 12 Months after Index Date		12 months
Proportion of Patients with Treatment-emergent Adverse Events (TEAEs), per Category	Categories were TEAEs, treatment-related TEAEs, serious TEAEs, TEAEs leading to discontinuation of siponimod, TEAEs leading to death, and TEAEs of special interest (AESIs). TEAEs were defined as any AEs that were not presented prior to medical treatment, or an already presented event that worsens either in intensity or frequency following the treatment, that developed after siponimod initiation.	Up to approximately 32 months
Proportion of Patients with Selected Notable Abnormal Laboratory Data	Selected notable abnormal laboratory data included complete blood count with differential lymphocyte count and liver function tests.	Up to approximately 32 months

Collaborators and Investigators

• Sponsor: Novartis
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): April 23, 2023
• Primary Completion (Actual): August 31, 2023
• Study Completion (Actual): August 31, 2023

Study Registration Dates

• First Submitted: April 28, 2024
• First Submitted That Met QC Criteria: May 1, 2024
• First Posted (Actual): May 2, 2024

Study Record Updates

• Last Update Posted (Actual): May 2, 2024
• Last Update Submitted That Met QC Criteria: May 1, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Disease Attributes; Chronic Disease; Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive; Sclerosis; Multiple Sclerosis, Relapsing-Remitting
• Other Study ID Numbers: CBAF312A2413

Drug and device information, study documents

• product manufactured in and exported from the U.S.: Yes