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

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02555215

Enrollment

Registered

2015-09-21

Start date

2016-02-22

Completion date

2018-09-24

Last updated

2019-11-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Multiple Sclerosis, Relapsing-Remitting

Keywords

Pediatrics

Brief summary

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).

Interventions

DRUGdimethyl fumarate

administered orally

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

10 Years to 17 Years

Healthy volunteers

Inclusion criteria

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/

Design outcomes

Primary

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

Secondary

Measure	Time frame	Description
Average Annualized Relapse Rate (ARR)	Baseline to Week 96	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.
Percentage of Participants Experiencing One or More Relapses	Baseline to Week 96	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.
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24	Week 16 to Week 24	T2 hyperintense lesions were measured by MRI brain scans.
Number of Participants Experiencing Disability Progression	Baseline to Week 96	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.
Change From Baseline in the Degree of Disability	Baseline to Week 96	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.
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72	Week 64 to Week 72	T2 hyperintense lesions were measured by MRI brain scans.

Countries

Belgium, Bulgaria, Czechia, Germany, Kuwait, Latvia, Lebanon, Poland, Turkey (Türkiye), United States

Participant flow

Participants by arm

Arm	Count
Dimethyl Fumarate Participants will receive 120 mg capsule(s) taken orally.	20
Total	20

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Investigator Decision	2
Overall Study	Other	1

Baseline characteristics

Characteristic	Dimethyl Fumarate
Age, Categorical <=18 years	14 Participants
Age, Categorical >=65 years	0 Participants
Age, Categorical Between 18 and 65 years	6 Participants
Age, Continuous	16.7 years STANDARD_DEVIATION 1.31
Race/Ethnicity, Customized Asian	1 participants
Race/Ethnicity, Customized Black or African American	0 participants
Race/Ethnicity, Customized Not Reported Due to Confidentiality Regulations	13 participants
Race/Ethnicity, Customized Other	1 participants
Race/Ethnicity, Customized White	5 participants
Sex: Female, Male Female	13 Participants
Sex: Female, Male Male	7 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 20
other Total, other adverse events	18 / 20
serious Total, serious adverse events	2 / 20

Outcome results

Primary

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.