Study of Efficacy and Safety of Fingolimod (Gilenya) 0.5 mg in Chinese Patients With Relapsing Multiple Sclerosis (RMS) Patients

A 24-month, Open-label, Prospective, Multicenter Interventional, Single-arm Study Assessing the Efficacy and Safety of Fingolimod (Gilenya) 0.5 mg in Relapsing Multiple Sclerosis (RMS) Patients in China

To assess the efficacy and safety of 0.5mg Fingolimod (Gilenya) in Chinese patients with relapsing relapsing multiple sclerosis (RMS)

Study Overview

• Status: Active, not recruiting
• Conditions: Relapsing Multiple Sclerosis (RMS)
• Intervention / Treatment: Drug: Fingolimod 0.5mg

Detailed Description

This is a 24-month, open-label, multicenter, interventional, single-arm study to collect efficacy and, safety of oral fingolimod 0.5 mg/day in approximately 100 relapsing multiple sclerosis (RMS) subjects in China.

The study will consist of three Phases:

Screening (up to 1 month): After signing informed consent, subjects will enter a Screening Phase to determine eligibility according to inclusion and exclusion criteria.

Treatment Period (24 months): On visit Day 1, all eligibility criteria will be confirmed, including a pre-dose ECG and vital signs. The first dose of study drug will be taken in the clinic on Day 1 and the subject will be monitored for 6 hours after the first dose administration before discharge. Participants will return to site for evaluation at month 1 and then every three months until the end of treatment up to 24 months.

Follow Up (2 months): Subjects who completed Treatment Period or discontinued from treatment will return for the Follow-up visit 2 months after the last dose of study drug.

• Study Type: Interventional
• Enrollment (Actual): 98
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100028
    • Novartis Investigative Site
  • Guang Zhou, China, 510260
    • Novartis Investigative Site
  • Shanghai, China, 200040
    • Novartis Investigative Site
  • Beijing
    • Beijing, Beijing, China, 100000
      • Novartis Investigative Site
    • Beijing, Beijing, China, 065001
      • Novartis Investigative Site
  • Guangdong
    • Guangzhou, Guangdong, China, 510630
      • Novartis Investigative Site
    • Guangzhou, Guangdong, China, 510623
      • Novartis Investigative Site
  • Henan
    • Zhengzhou, Henan, China, 450052
      • Novartis Investigative Site
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Novartis Investigative Site
  • Jiangsu
    • Suzhou, Jiangsu, China, 215004
      • Novartis Investigative Site
  • Jilin
    • Changchun, Jilin, China, 130021
      • Novartis Investigative Site
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • Novartis Investigative Site
  • Zhejiang
    • Wenzhou, Zhejiang, China, 325000
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participant 10 to 17 years old inclusive with weight > 40kg.
• Participant 18 to 65 years old inclusive;
• Participants with relapsing multiple sclerosis
• Participants never used fingolimod before enrollment
• Subjects with Expanded Disability Status Scale (EDSS) score of 0 - 6.0 (inclusive) at Screening

Exclusion Criteria:

• Participants with certain cardiovascular conditions and/or findings in the screening ECG.
• Diagnosis of macular edema during screening visit.
• Increased risk for opportunistic infections
• Participants with known active malignancies.
• Participants who have been treated with teriflunomide within 3.5 months prior to baseline, except if active washout.
• Participants with severe active infections, active chronic infection.
• Participants with severe liver impairment.
• Pregnant confirmed by a positive pregnancy test or nursing (lactating) women.

Other protocol-specified inclusion or exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Fingolimod; Fingolimod 0.5 mg capsule taken orally once daily	Drug: Fingolimod 0.5mg; Subjects will receive fingolimod 0.5mg capsule QD up to month 24; Other Names: FTY720

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in Annualized relapse rate(ARR)	A relapse is an appearance of a new neurological abnormality or worsening of previously stable or improving pre-existing neurological abnormality, separated by at least 30 days from onset of a preceding clinical demyelinating event which is present for at least 24 hours in the absence of fever or infection. A confirmed relapse by Treating Physician must be confirmed within 7 days of onset of symptoms and accompanied by an increase of at least half a step (0.5) on the Expanded Disability Status Scale (EDSS) or an increase of 1 point on two different Functional Systems (FS) of the EDSS or 2 points on one of the FS.The EDSS is an ordinal scale used for assessing neurologic impairment based on an exam consisting of seven functional systems (FSs)which are scored from 0 to 10 (death from MS) and an ambulation score that are combined to determine the EDSS steps. The higher score means the worsening of neurological status.	Baseline to Month 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of Adverse events (AE) and serious adverse events (SAE)	Adverse events will be collected at each visit throughout the trial including a 2 month follow up period. Results from safety assessments may be recorded as adverse events if determined by the investigator to meet requirements of clinical significance and meeting definition of adverse event	Baseline up to Month 26
Change from baseline in T1 hypo-intense lesion volume	T1 hypo-intense lesions as measured by Magnetic Resonance Imaging (MRI)	Baseline up to Month 24
Change from baseline in T2 lesion-new/newly enhancing lesion volume	T2 lesion-new/newly enhancing lesions as measured by MRI	Baseline up to Month 24
Change from baseline in Gd-enhancing T1 lesion volume	Gd-enhancing T1 lesions as measured by MRI	Baseline up to Month 24
Change from baseline in number of T1 hypo-intense lesions	Number of T1 hypo-intense lesions as measured by MRI	Baseline up to Month 24
Change from baseline in number of T2 lesions-new/newly enhancing lesions	Number of T2 lesions-new/newly enhancing lesions as measured by MRI	Baseline up to Month 24
Change from baseline in number of Gd-enhancing T1 lesions	Number of Gd-enhancing T1 lesions as measured by MRI	Baseline up to Month 24

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): February 20, 2021
• Primary Completion (Estimated): March 28, 2025
• Study Completion (Estimated): March 28, 2025

Study Registration Dates

• First Submitted: December 8, 2020
• First Submitted That Met QC Criteria: December 8, 2020
• First Posted (Actual): December 16, 2020

Study Record Updates

• Last Update Posted (Actual): October 3, 2023
• Last Update Submitted That Met QC Criteria: September 29, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: China; Pediatric; Adult; Fingolimod; Relapse Multiple Sclerosis (RMS)
• 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; Multiple Sclerosis; Sclerosis; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Immunosuppressive Agents; Immunologic Factors; Sphingosine 1 Phosphate Receptor Modulators; Fingolimod Hydrochloride
• Other Study ID Numbers: CFTY720D2419

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No