Extension Study of BG00012 in Pediatric Subjects With Relapsing Remitting Multiple Sclerosis (RRMS)

Study Overview
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Brief Summary
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The primary objective of the study is to evaluate the long-term safety of BG00012 in subjects who completed Study 109MS202 (NCT02410200). Secondary objectives are as follows: To evaluate the long-term efficacy of BG00012 and to describe the long-term Multiple Sclerosis (MS) outcomes in subjects who completed Study 109MS202 (NCT02410200).

Official Title
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A Multicenter Extension Study to Determine the Long-Term Safety and Efficacy of BG00012 in Pediatric Subjects With Relapsing-Remitting Multiple Sclerosis

Conditions
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Multiple Sclerosis, Relapsing-Remitting

Intervention / Treatment
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* Drug: dimethyl fumarate


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Ability of parents, legal guardians, and/or subjects to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local subject privacy regulations. Subjects will provide assent in addition to the parental or guardian consent, as appropriate, per local regulations. Subjects who completed, as per protocol, the previous BG00012 clinical study 109MS202 (NCT02410200) and remain on BG00012 treatment. Key Exclusion Criteria: Unwillingness or inability to comply with study requirements, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to comply with the protocol. Any significant changes in medical history occurring after enrollment in the parent Study 109MS202 (NCT02410200), including laboratory test abnormalities or current clinically significant conditions that in the opinion of the Investigator would have excluded the subject's participation from the parent study. The Investigator must re-review the subject's medical fitness for participation and consider any factors that would preclude treatment. Subjects from Study 109MS202 (NCT02410200) who could not tolerate study treatment. NOTE: Other protocol defined inclusion/exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 10 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: dimethyl fumarate<br>Participants will receive 120 mg capsule(s) taken orally. | Drug: dimethyl fumarate<br>* administered orally<br>* Other names: BG00012;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE is any untoward medical occurrence that does not necessarily have a causal relationship with treatment. An SAE is 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. | Baseline to Week 96 | 
| Number of Participants Discontinuing Treatment Due to an Adverse Event | An AE is any untoward medical occurrence that does not necessarily have a causal relationship with treatment. | Baseline to Week 96 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24 | T2 hyperintense lesions were measured by MRI brain scans. | Week 16 to Week 24 | 
| Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72 | T2 hyperintense lesions were measured by MRI brain scans. | Week 64 to Week 72 | 
| Average Annualized Relapse Rate (ARR) | Relapses were defined as new or recurrent neurologic symptoms not associated with fever or infection, lasting at least 24 hours, and accompanied by new objective neurological findings upon examination by the Investigator. New or recurrent neurologic symptoms that evolved gradually over months were considered disability progression, not an acute relapse, and were not treated with steroids. The ARR was calculated as the total number of relapses that occurred during the previous 12 months and during the 120 weeks on treatment for participants in Study 109MS202 that continued into Study 109MS311, divided by the total number of person-years followed prior to the study and by the total number of person-years followed during the study, respectively. | Baseline to Week 96 | 
| Percentage of Participants Experiencing One or More Relapses | Relapses were defined as new or recurrent neurologic symptoms not associated with fever or infection, lasting at least 24 hours, and accompanied by new objective neurological findings upon examination by the Investigator. New or recurrent neurologic symptoms that evolved gradually over months were considered disability progression, not an acute relapse, and were not treated with steroids. | Baseline to Week 96 | 
| Change From Baseline in the Degree of Disability | The Expanded Disability Status Scale (EDSS) measures disability status on a scale ranging from 0 to 10, with higher scores indicating more disability. Scoring is based on measures of impairment in eight functional systems on examination by a neurologist. | Baseline to Week 96 | 
| Number of Participants Experiencing Disability Progression | Measured by at least a 1.0-point increase on the EDSS from baseline EDSS ≥1.0 that is sustained for 24 weeks, or at least a 1.5-point increase on the EDSS from baseline EDSS = 0 that is sustained for 24 weeks. | Baseline to Week 96 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pediatrics