Clinical trial NCT03870763: 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
Overall status: TERMINATED
Phase: PHASE3
Conditions: Multiple Sclerosis, Relapsing-Remitting
Keywords: Pediatric, Multiple Sclerosis
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2019-03-12
Enrollment: 11
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Time to First Relapse
timeFrame: Baseline up to Week 96
Secondary outcomes:
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to Week 100
measure: Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans at Weeks 48 and 96
timeFrame: Weeks 48 and 96
measure: Number of Galdolinium (Gd)-Enhancing Lesions at Weeks 48 and 96
timeFrame: Weeks 48 and 96
measure: Annualized Relapse Rate
timeFrame: Up to Week 96
Other outcomes:
Locations:
city: Raleigh
country: United States
facility: Research Site
state: North Carolina
city: Medellín
country: Colombia
facility: Research Site
city: Tallinn
country: Estonia
facility: Research Site
city: Budapest
country: Hungary
facility: Research Site
city: Ar-Ramtha
country: Jordan
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Petaling Jaya
country: Malaysia
facility: Research Site
city: Seberang Jaya
country: Malaysia
facility: Research Site
city: Guadalajara
country: Mexico
facility: Research Site
city: Morelia
country: Mexico
facility: Research Site
city: Santa Cruz
country: Mexico
facility: Research Site
city: Dammam
country: Saudi Arabia
facility: Research Site
city: Riyadh
country: Saudi Arabia
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
city: Taoyuan
country: Taiwan
facility: Research Site
city: Bangkok
country: Thailand
facility: Research Site
city: Manouba
country: Tunisia
facility: Research Site
city: Monastir
country: Tunisia
facility: Research Site
city: Sfax
country: Tunisia
facility: Research Site
city: Tunis
country: Tunisia
facility: Research Site
city: Ankara
country: Turkey
facility: Research Site
city: Izmir
country: Turkey
facility: Research Site
city: Samsun
country: Turkey
facility: Research Site
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: