Study to Evaluate the Efficacy and Safety of Dimethyl Fumarate (Tecfidera) and Peginterferon Beta-1a (Plegridy) for the Treatment of Relapsing-Remitting Multiple Sclerosis in Pediatric Participants

Multiple Sclerosis, Relapsing-Remitting Clinical Trial

Official title:

A Randomized, Double-Blind, Double-Dummy, Placebo-Controlled, 3-Arm, Parallel Group Study in Pediatric Subjects Aged 10 Through 17 Years to Evaluate the Efficacy and Safety of BG00012 and BIIB017 for the Treatment of Relapsing-Remitting Multiple Sclerosis

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03870763
• Other study ID #: 800MS301
• Secondary ID: 2018-000516-22
• Status: Terminated
• Phase: Phase 3
• Start date: March 19, 2019
• Est. completion date: July 21, 2022

Study information

• Verified date: May 2023
• Source: Biogen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The main objective of the study is to evaluate the efficacy of dimethyl fumarate (Tecfidera) and peginterferon beta-1a (Plegridy), both compared with placebo, in pediatric participants with RRMS. The other objectives of this study are to evaluate the safety and tolerability of dimethyl fumarate and peginterferon beta-1a and to assess the effect of dimethyl fumarate and peginterferon beta-1a, both compared with placebo, on additional clinical and radiological measures of disease activity.

Description:

Participants will be randomized in a 1:2:2 ratio to receive the double-blind study treatment (Dimethyl Fumarate, Peginterferon Beta-1a, and placebo). Participants experiencing a confirmed relapse or disability progression or high lesion burden on MRI will have the option to discontinue the blinded study treatment and switch to an alternative therapy or open-label BG00012.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 11
• Est. completion date: July 21, 2022
• Est. primary completion date: July 21, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years to 17 Years

Eligibility

Key Inclusion Criteria:

• Must have a diagnosis of RRMS as defined by the revised consensus definition for pediatric MS
• Must have an EDSS score between 0.0 and 5.0.
• Must have a body weight of =30 kg
• Must have experienced =1 relapse in the 12 months prior to randomization (Day 1), or must have evidence of asymptomatic disease activity seen on MRI in the 6 months prior to randomization, or =2 relapses in the 24 months prior to randomization (Day 1). Relapse is defined as the occurrence of a clinical demyelination event regardless of whether the event is a first or subsequent demyelinating event.

Key Exclusion Criteria:

• Participants having primary progressive, secondary progressive, or progressive RMS.
• Disorders mimicking MS, such as other demyelinating disorders, systemic autoimmune disorders, metabolic disorders, and infectious disorders.
• History of clinically significant cardiovascular, pulmonary, GI, hepatic, renal, endocrinologic, hematologic, immunologic, metabolic, dermatologic, growth, developmental, psychiatric (including depression), neurologic (other than MS), and/or other major disease and/or laboratory abnormality indicative thereof, that would preclude participation in a clinical study
• Occurrence of an MS relapse within the 30 days prior to randomization (Day 1) and/or the subject has not stabilized from a previous relapse prior to randomization NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Multiple Sclerosis
• Multiple Sclerosis, Relapsing-Remitting
• Sclerosis

Intervention

Drug:
• Dimethyl Fumarate
Administered as specified in the treatment arm.
• Peginterferon Beta-1a
Administered as specified in the treatment arm.
• Placebo
Administered as specified in the treatment arm.

Locations

Country	Name	City	State
Colombia	Research Site	Medellín
Estonia	Research Site	Tallinn
Hungary	Research Site	Budapest
Jordan	Research Site	Ar-Ramtha
Korea, Republic of	Research Site	Seoul
Malaysia	Research Site	Petaling Jaya
Malaysia	Research Site	Seberang Jaya
Mexico	Research Site	Guadalajara
Mexico	Research Site	Morelia
Mexico	Research Site	Santa Cruz
Saudi Arabia	Research Site	Dammam
Saudi Arabia	Research Site	Riyadh
Taiwan	Research Site	Taipei
Taiwan	Research Site	Taoyuan
Thailand	Research Site	Bangkok
Tunisia	Research Site	Manouba
Tunisia	Research Site	Monastir
Tunisia	Research Site	Sfax
Tunisia	Research Site	Tunis
Turkey	Research Site	Ankara
Turkey	Research Site	Izmir
Turkey	Research Site	Samsun
United States	Research Site	Raleigh	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Countries where clinical trial is conducted

United States,  Colombia,  Estonia,  Hungary,  Jordan,  Korea, Republic of,  Malaysia,  Mexico,  Saudi Arabia,  Taiwan,  Thailand,  Tunisia,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to First Relapse	A clinical relapse is defined as new or recurrent neurological symptoms, not associated with fever, lasting for at least 24 hours, and followed by a period of 30 days of stability or improvement. Time to relapse is defined as from the first dose of the study drug to the day of relapse. Time to First Relapse is estimated by Kaplan Mayer method.	Baseline up to Week 96
Secondary	Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; Initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.	Baseline up to Week 100
Secondary	Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans at Weeks 48 and 96	The number of new or newly enlarging T2 hyperintense lesions that developed in each participant assessed on magnetic resonance imaging (MRI) scans.	Weeks 48 and 96
Secondary	Number of Galdolinium (Gd)-Enhancing Lesions at Weeks 48 and 96	The number of Gd-enhancing lesions was assessed by using MRI scans.	Weeks 48 and 96
Secondary	Annualized Relapse Rate	A clinical relapse was defined as new or recurrent neurological symptoms, not associated with fever, lasting for at least 24 hours, and followed by a period of 30 days of stability or improvement. The relapse rate for an individual participant was calculated as the number of relapses for that participant divided by the number of participant-years followed. The ARR for each enrolment group was calculated as the total number of relapses experienced in the group divided by the total number of participant-years on the study. An unadjusted relapse rate is reported.	Up to Week 96