Back to resultsStudy 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
Primary Purpose
Multiple Sclerosis, Relapsing-Remitting
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Dimethyl Fumarate
Peginterferon Beta-1a
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Multiple Sclerosis, Relapsing-Remitting focused on measuring Pediatric, Multiple Sclerosis
Eligibility Criteria
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.
Sites / Locations
- Research Site
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Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
Dimethyl Fumarate 240 mg
Peginterferon Beta-1a 125 µg
Placebo
Arm Description
Participants will receive dimethyl fumarate 240 milligrams (mg) capsule twice daily (BID) orally and placebo subcutaneous (SC) injection every 2 weeks for up to 96 weeks (2 years).
Participants will receive peginterferon beta-1a 125 micrograms (µg) SC injection every 2 weeks and placebo capsule BID orally for up to 96 weeks (2 years).
Participants will receive placebo SC injection every 2 weeks and placebo capsule BID orally for up to 96 weeks (2 years)
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
Number of Galdolinium (Gd)-Enhancing Lesions at Weeks 48 and 96
The number of Gd-enhancing lesions was assessed by using MRI scans.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03870763
Brief Title
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
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Terminated
Why Stopped
Decision to stop the trial was based on long-term difficulties in fulfilling our enrolment commitments and changes in paediatric MS landscape which no longer support placebo-controlled trials.Decision to stop study was not based on safety concerns.
Study Start Date
March 19, 2019 (Actual)
Primary Completion Date
July 21, 2022 (Actual)
Study Completion Date
July 21, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple Sclerosis, Relapsing-Remitting
Keywords
Pediatric, Multiple Sclerosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dimethyl Fumarate 240 mg
Arm Type
Experimental
Arm Description
Participants will receive dimethyl fumarate 240 milligrams (mg) capsule twice daily (BID) orally and placebo subcutaneous (SC) injection every 2 weeks for up to 96 weeks (2 years).
Arm Title
Peginterferon Beta-1a 125 µg
Arm Type
Experimental
Arm Description
Participants will receive peginterferon beta-1a 125 micrograms (µg) SC injection every 2 weeks and placebo capsule BID orally for up to 96 weeks (2 years).
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive placebo SC injection every 2 weeks and placebo capsule BID orally for up to 96 weeks (2 years)
Intervention Type
Drug
Intervention Name(s)
Dimethyl Fumarate
Other Intervention Name(s)
Tecfidera, BG00012
Intervention Description
Administered as specified in the treatment arm.
Intervention Type
Drug
Intervention Name(s)
Peginterferon Beta-1a
Other Intervention Name(s)
Plegridy, BIIB017
Intervention Description
Administered as specified in the treatment arm.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Administered as specified in the treatment arm.
Primary Outcome Measure Information:
Title
Time to First Relapse
Description
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.
Time Frame
Baseline up to Week 96
Secondary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description
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.
Time Frame
Baseline up to Week 100
Title
Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans at Weeks 48 and 96
Description
The number of new or newly enlarging T2 hyperintense lesions that developed in each participant assessed on magnetic resonance imaging (MRI) scans.
Time Frame
Weeks 48 and 96
Title
Number of Galdolinium (Gd)-Enhancing Lesions at Weeks 48 and 96
Description
The number of Gd-enhancing lesions was assessed by using MRI scans.
Time Frame
Weeks 48 and 96
Title
Annualized Relapse Rate
Description
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.
Time Frame
Up to Week 96
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Raleigh
State/Province
North Carolina
ZIP/Postal Code
27607
Country
United States
Facility Name
Research Site
City
Medellín
Country
Colombia
Facility Name
Research Site
City
Tallinn
ZIP/Postal Code
11315
Country
Estonia
Facility Name
Research Site
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
Research Site
City
Ar-Ramtha
Country
Jordan
Facility Name
Research Site
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Research Site
City
Petaling Jaya
Country
Malaysia
Facility Name
Research Site
City
Seberang Jaya
Country
Malaysia
Facility Name
Research Site
City
Guadalajara
Country
Mexico
Facility Name
Research Site
City
Morelia
Country
Mexico
Facility Name
Research Site
City
Santa Cruz
Country
Mexico
Facility Name
Research Site
City
Dammam
Country
Saudi Arabia
Facility Name
Research Site
City
Riyadh
Country
Saudi Arabia
Facility Name
Research Site
City
Taipei
ZIP/Postal Code
10002
Country
Taiwan
Facility Name
Research Site
City
Taoyuan
ZIP/Postal Code
333
Country
Taiwan
Facility Name
Research Site
City
Bangkok
Country
Thailand
Facility Name
Research Site
City
Manouba
Country
Tunisia
Facility Name
Research Site
City
Monastir
Country
Tunisia
Facility Name
Research Site
City
Sfax
Country
Tunisia
Facility Name
Research Site
City
Tunis
Country
Tunisia
Facility Name
Research Site
City
Ankara
Country
Turkey
Facility Name
Research Site
City
Izmir
Country
Turkey
Facility Name
Research Site
City
Samsun
Country
Turkey
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
IPD Sharing URL
https://vivli.org/
Learn more about this trial
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
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