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Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With RRMS (FOCUS)

Primary Purpose

Multiple Sclerosis, Relapsing-Remitting

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
dimethyl fumarate
Sponsored by
Biogen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Multiple Sclerosis, Relapsing-Remitting focused on measuring Pediatrics

Eligibility Criteria

10 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Research Site
  • Research Site
  • Research Site
  • Research Site
  • Research Site
  • Research Site
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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BG00012

Arm Description

Oral BG00012 120 mg twice daily (BID) for the first 7 days followed by 240 mg BID for 24 weeks.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Maximum Observed Plasma Concentration (Cmax)
Time to Reach Maximum Observed Plasma Concentration (Tmax)
Apparent Clearance (CL/F)
Apparent Volume of Distribution (V/F)
Half-Life Lambda z
Area Under the Concentration-Time Curve From Time 0 to Infinity (AUC0-inf)
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
AE: any untoward medical occurrence that does not necessarily have a causal relationship with treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above.

Full Information

First Posted
April 2, 2015
Last Updated
September 21, 2017
Sponsor
Biogen
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1. Study Identification

Unique Protocol Identification Number
NCT02410200
Brief Title
Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With RRMS
Acronym
FOCUS
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

2. Study Status

Record Verification Date
September 2017
Overall Recruitment Status
Completed
Study Start Date
September 30, 2015 (Actual)
Primary Completion Date
September 23, 2016 (Actual)
Study Completion Date
September 23, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple Sclerosis, Relapsing-Remitting
Keywords
Pediatrics

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
22 (Actual)

8. Arms, Groups, and Interventions

Arm Title
BG00012
Arm Type
Experimental
Arm Description
Oral BG00012 120 mg twice daily (BID) for the first 7 days followed by 240 mg BID for 24 weeks.
Intervention Type
Drug
Intervention Name(s)
dimethyl fumarate
Other Intervention Name(s)
BG00012, DMF
Primary Outcome Measure Information:
Title
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
Time Frame
Baseline Period (Week -8 to Day 0), On-Treatment Assessment Period (Week 16 to Week 24)
Secondary Outcome Measure Information:
Title
Maximum Observed Plasma Concentration (Cmax)
Time Frame
Day 8
Title
Time to Reach Maximum Observed Plasma Concentration (Tmax)
Time Frame
Day 8
Title
Apparent Clearance (CL/F)
Time Frame
Day 8
Title
Apparent Volume of Distribution (V/F)
Time Frame
Day 8
Title
Half-Life Lambda z
Time Frame
Day 8
Title
Area Under the Concentration-Time Curve From Time 0 to Infinity (AUC0-inf)
Time Frame
Day 8
Title
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description
AE: any untoward medical occurrence that does not necessarily have a causal relationship with treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above.
Time Frame
Up to Week 28

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: 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.
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
San Bernardino
State/Province
California
ZIP/Postal Code
92408
Country
United States
Facility Name
Research Site
City
Gent
ZIP/Postal Code
B-9000
Country
Belgium
Facility Name
Research Site
City
Sofia
ZIP/Postal Code
B-1113
Country
Bulgaria
Facility Name
Research Site
City
Hradec kralove
ZIP/Postal Code
500 05
Country
Czechia
Facility Name
Research Site
City
Munchen
State/Province
Bayern
ZIP/Postal Code
80337
Country
Germany
Facility Name
Research Site
City
Gottingen
State/Province
Niedersachsen
ZIP/Postal Code
37075
Country
Germany
Facility Name
Research Site
City
Dasman
State/Province
Kuwait City
ZIP/Postal Code
15462
Country
Kuwait
Facility Name
Research Site
City
Riga
ZIP/Postal Code
LV-1004
Country
Latvia
Facility Name
Research Site
City
Beirut
ZIP/Postal Code
1107 2020
Country
Lebanon
Facility Name
Research Site
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Research Site
City
Poznan
ZIP/Postal Code
60-355
Country
Poland
Facility Name
Research Site
City
Ankara
ZIP/Postal Code
06100
Country
Turkey

12. IPD Sharing Statement

Citations:
PubMed Identifier
29681490
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.
Results Reference
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Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With RRMS

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