Clinical trial NCT02410200: Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With RRMS
Official title: Open-Label, Multicenter, Multiple-Dose Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With Relapsing-Remitting Multiple Sclerosis Aged 10 to 17 Years
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multiple Sclerosis, Relapsing-Remitting
Keywords: Pediatrics
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2015-04-07
Enrollment: 22
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to evaluate the effect of BG00012 (dimethyl fumarate) on brain magnetic resonance imaging (MRI) lesions in pediatric participants with relapsing-remitting multiple sclerosis (RRMS). The secondary objectives of this study are to characterize the pharmacokinetics of BG00012 in pediatric participants with RRMS and to evaluate the safety and tolerability of BG00012 in pediatric participants with RRMS.


Eligibility criteria: Key Inclusion Criteria:

* Ability of parents or legal guardians to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations. Subjects will provide assent in addition to the parent or legal guardian, as appropriate, as per local regulations.
* Must have a body weight of ≥30 kg at Screening and Day 1.
* Must have a diagnosis of RRMS according to McDonald criteria for MS (2010) \[Polman 2011\] and International Pediatric Multiple Sclerosis (MS) Study Group criteria for pediatric MS (2013) \[Krupp 2013\].

Key Exclusion Criteria:

* Primary progressive, secondary progressive, or progressive relapsing MS (as defined by \[Lublin and Reingold 1996\]). These conditions require the presence of continuous clinical disease worsening over a period of at least 3 months. Subjects with these conditions may also have superimposed relapses but are distinguished from relapsing-remitting subjects by the lack of clinically stable periods or clinical improvement.
* Disorders mimicking MS, such as other demyelinating disorders (e.g., acute disseminated encephalomyelitis), systemic autoimmune disorders (e.g., Sjögren disease, lupus erythematosus, and neuromyelitis optica), metabolic disorders (e.g., dystrophies), and infectious disorders.
* History of severe allergic or anaphylactic reactions or known drug hypersensitivity to dimethyl fumarate or fumaric acid esters.

NOTE: Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Change in the Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans From the Baseline Period to On-Treatment Assessment Period
timeFrame: Baseline Period (Week -8 to Day 0), On-Treatment Assessment Period (Week 16 to Week 24)
Secondary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax)
timeFrame: Day 8
measure: Time to Reach Maximum Observed Plasma Concentration (Tmax)
timeFrame: Day 8
measure: Apparent Clearance (CL/F)
timeFrame: Day 8
measure: Apparent Volume of Distribution (V/F)
timeFrame: Day 8
measure: Half-Life Lambda z
timeFrame: Day 8
measure: Area Under the Concentration-Time Curve From Time 0 to Infinity (AUC0-inf)
timeFrame: Day 8
measure: Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Up to Week 28
Other outcomes:
Locations:
city: San Bernardino
country: United States
facility: Research Site
state: California
city: Gent
country: Belgium
facility: Research Site
city: Sofia
country: Bulgaria
facility: Research Site
city: Hradec kralove
country: Czechia
facility: Research Site
city: Munchen
country: Germany
facility: Research Site
state: Bayern
city: Gottingen
country: Germany
facility: Research Site
state: Niedersachsen
city: Dasman
country: Kuwait
facility: Research Site
state: Kuwait City
city: Riga
country: Latvia
facility: Research Site
city: Beirut
country: Lebanon
facility: Research Site
city: Gdansk
country: Poland
facility: Research Site
city: Poznan
country: Poland
facility: Research Site
city: Ankara
country: Turkey
facility: Research Site
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: Alroughani R, Das R, Penner N, Pultz J, Taylor C, Eraly S. Safety and Efficacy of Delayed-Release Dimethyl Fumarate in Pediatric Patients With Relapsing Multiple Sclerosis (FOCUS). Pediatr Neurol. 2018 Jun;83:19-24. doi: 10.1016/j.pediatrneurol.2018.03.007. Epub 2018 Mar 22.