Trial Outcomes & Findings for Extension Study of BG00012 in Pediatric Subjects With Relapsing Remitting Multiple Sclerosis (RRMS) (NCT NCT02555215)
NCT ID: NCT02555215
Last Updated: 2019-11-22
Results Overview
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.
COMPLETED
PHASE3
20 participants
Baseline to Week 96
2019-11-22
Participant Flow
Participant milestones
| Measure |
Dimethyl Fumarate
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Dimethyl Fumarate
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Overall Study
Other
|
1
|
|
Overall Study
Investigator Decision
|
2
|
Baseline Characteristics
Extension Study of BG00012 in Pediatric Subjects With Relapsing Remitting Multiple Sclerosis (RRMS)
Baseline characteristics by cohort
| Measure |
Dimethyl Fumarate
n=20 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Age, Categorical
<=18 years
|
14 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
16.7 years
STANDARD_DEVIATION 1.31 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
5 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Reported Due to Confidentiality Regulations
|
13 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 96Population: The safety population was defined as all participants who received at least 1 dose of BG00012.
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.
Outcome measures
| Measure |
Dimethyl Fumarate
n=20 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
AE
|
18 participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
SAE
|
2 participants
|
PRIMARY outcome
Timeframe: Baseline to Week 96An AE is any untoward medical occurrence that does not necessarily have a causal relationship with treatment.
Outcome measures
| Measure |
Dimethyl Fumarate
n=20 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Number of Participants Discontinuing Treatment Due to an Adverse Event
|
0 participants
|
SECONDARY outcome
Timeframe: Week 16 to Week 24Population: Participants who received at least 1 dose of BG00012 in this study and had an evaluation of the efficacy endpoint under analysis.
T2 hyperintense lesions were measured by MRI brain scans.
Outcome measures
| Measure |
Dimethyl Fumarate
n=17 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24
0 lesions
|
12 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24
1 lesion
|
2 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24
2 lesions
|
1 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24
3 lesions
|
1 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24
4 lesions
|
0 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 16 to Week 24
5 or more lesions
|
1 Participants
|
SECONDARY outcome
Timeframe: Week 64 to Week 72Population: Participants who received at least 1 dose of BG00012 in this study and had an evaluation of the efficacy endpoint under analysis.
T2 hyperintense lesions were measured by MRI brain scans.
Outcome measures
| Measure |
Dimethyl Fumarate
n=10 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72
0 lesions
|
8 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72
1 lesion
|
1 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72
2 lesions
|
1 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72
3 lesions
|
0 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72
4 lesions
|
0 Participants
|
|
Total Number of New or Newly Enlarging T2 Hyperintense Lesions From Week 64 to Week 72
5 or more lesions
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 96Population: Participants who received at least 1 dose of BG00012 in this study and had an evaluation of the efficacy endpoint under analysis.
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.
Outcome measures
| Measure |
Dimethyl Fumarate
n=20 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Average Annualized Relapse Rate (ARR)
|
0.1 relapses per person-years
|
SECONDARY outcome
Timeframe: Baseline to Week 96Relapses 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.
Outcome measures
| Measure |
Dimethyl Fumarate
n=20 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Percentage of Participants Experiencing One or More Relapses
|
10 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline to Week 96Population: Participants who received at least 1 dose of BG00012 in this study and had an evaluation of the efficacy endpoint under analysis.
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.
Outcome measures
| Measure |
Dimethyl Fumarate
n=20 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Change From Baseline in the Degree of Disability
Baseline
|
1.00 score on a scale
Standard Deviation 1.026
|
|
Change From Baseline in the Degree of Disability
Change from Baseline at Week 12
|
0.15 score on a scale
Standard Deviation 0.718
|
|
Change From Baseline in the Degree of Disability
Change from Baseline at Week 24
|
0.29 score on a scale
Standard Deviation 0.508
|
|
Change From Baseline in the Degree of Disability
Change from Baseline at Week 36
|
0.27 score on a scale
Standard Deviation 0.832
|
|
Change From Baseline in the Degree of Disability
Change from Baseline at Week 48
|
0.50 score on a scale
Standard Deviation 0.791
|
|
Change From Baseline in the Degree of Disability
Change from Baseline at Week 72
|
0.71 score on a scale
Standard Deviation 1.010
|
|
Change From Baseline in the Degree of Disability
Change from Baseline at Week 96
|
0.21 score on a scale
Standard Deviation 0.964
|
SECONDARY outcome
Timeframe: Baseline to Week 96Population: Participants who received at least 1 dose of BG00012 in this study and had an evaluation of the efficacy endpoint under analysis.
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.
Outcome measures
| Measure |
Dimethyl Fumarate
n=20 Participants
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Number of Participants Experiencing Disability Progression
|
3 Participants
|
Adverse Events
BG00012
Serious adverse events
| Measure |
BG00012
n=20 participants at risk
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
5.0%
1/20 • Baseline to Week 96
|
|
Nervous system disorders
Multiple sclerosis relapse
|
5.0%
1/20 • Baseline to Week 96
|
Other adverse events
| Measure |
BG00012
n=20 participants at risk
Participants will receive 120 mg capsule(s) taken orally.
|
|---|---|
|
Ear and labyrinth disorders
Vertigo
|
10.0%
2/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Abdominal pain
|
15.0%
3/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Abdominal pain upper
|
10.0%
2/20 • Baseline to Week 96
|
|
General disorders
Fatigue
|
10.0%
2/20 • Baseline to Week 96
|
|
Infections and infestations
Gastroenteritis
|
10.0%
2/20 • Baseline to Week 96
|
|
Infections and infestations
Nasopharyngitis
|
10.0%
2/20 • Baseline to Week 96
|
|
Infections and infestations
Pharyngitis
|
10.0%
2/20 • Baseline to Week 96
|
|
Infections and infestations
Upper respiratory tract infection
|
15.0%
3/20 • Baseline to Week 96
|
|
Infections and infestations
Viral upper respiratory tract infection
|
15.0%
3/20 • Baseline to Week 96
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
10.0%
2/20 • Baseline to Week 96
|
|
Nervous system disorders
Headache
|
15.0%
3/20 • Baseline to Week 96
|
|
Nervous system disorders
Hypoaesthesia
|
10.0%
2/20 • Baseline to Week 96
|
|
Nervous system disorders
Multiple sclerosis relapse
|
20.0%
4/20 • Baseline to Week 96
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
15.0%
3/20 • Baseline to Week 96
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.0%
3/20 • Baseline to Week 96
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
10.0%
2/20 • Baseline to Week 96
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
10.0%
2/20 • Baseline to Week 96
|
|
Skin and subcutaneous tissue disorders
Acne
|
10.0%
2/20 • Baseline to Week 96
|
|
Vascular disorders
Flushing
|
25.0%
5/20 • Baseline to Week 96
|
|
Cardiac disorders
Wolff-parkinson-white syndrome
|
5.0%
1/20 • Baseline to Week 96
|
|
Eye disorders
Eye irritation
|
5.0%
1/20 • Baseline to Week 96
|
|
Eye disorders
Vision blurred
|
5.0%
1/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Abdominal discomfort
|
5.0%
1/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Diarrhoea
|
5.0%
1/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Dry mouth
|
5.0%
1/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Mouth ulceration
|
5.0%
1/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Nausea
|
5.0%
1/20 • Baseline to Week 96
|
|
Gastrointestinal disorders
Vomiting
|
5.0%
1/20 • Baseline to Week 96
|
|
General disorders
Influenza like illness
|
5.0%
1/20 • Baseline to Week 96
|
|
Immune system disorders
Dust allergy
|
5.0%
1/20 • Baseline to Week 96
|
|
Immune system disorders
Seasonal allergy
|
5.0%
1/20 • Baseline to Week 96
|
|
Immune system disorders
Smoke sensitivity
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Cystitis
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Herpes zoster
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Mastoiditis
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Respiratory tract infection viral
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Root canal infection
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Salpingo-oophoritis
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Sinusitis
|
5.0%
1/20 • Baseline to Week 96
|
|
Infections and infestations
Tonsillitis
|
5.0%
1/20 • Baseline to Week 96
|
|
Injury, poisoning and procedural complications
Burns second degree
|
5.0%
1/20 • Baseline to Week 96
|
|
Injury, poisoning and procedural complications
Fall
|
5.0%
1/20 • Baseline to Week 96
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
5.0%
1/20 • Baseline to Week 96
|
|
Injury, poisoning and procedural complications
Limb injury
|
5.0%
1/20 • Baseline to Week 96
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.0%
1/20 • Baseline to Week 96
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.0%
1/20 • Baseline to Week 96
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
5.0%
1/20 • Baseline to Week 96
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.0%
1/20 • Baseline to Week 96
|
|
Nervous system disorders
Demyelination
|
5.0%
1/20 • Baseline to Week 96
|
|
Nervous system disorders
Migraine
|
5.0%
1/20 • Baseline to Week 96
|
|
Nervous system disorders
Paraesthesia
|
5.0%
1/20 • Baseline to Week 96
|
|
Nervous system disorders
Presyncope
|
5.0%
1/20 • Baseline to Week 96
|
|
Nervous system disorders
Syncope
|
5.0%
1/20 • Baseline to Week 96
|
|
Psychiatric disorders
Irritability
|
5.0%
1/20 • Baseline to Week 96
|
|
Psychiatric disorders
Somatic symptom disorder
|
5.0%
1/20 • Baseline to Week 96
|
|
Renal and urinary disorders
Dysuria
|
5.0%
1/20 • Baseline to Week 96
|
|
Renal and urinary disorders
Hypertonic bladder
|
5.0%
1/20 • Baseline to Week 96
|
|
Renal and urinary disorders
Micturition urgency
|
5.0%
1/20 • Baseline to Week 96
|
|
Renal and urinary disorders
Urinary retention
|
5.0%
1/20 • Baseline to Week 96
|
|
Reproductive system and breast disorders
Menstruation irregular
|
5.0%
1/20 • Baseline to Week 96
|
|
Reproductive system and breast disorders
Ovarian cyst
|
5.0%
1/20 • Baseline to Week 96
|
|
Reproductive system and breast disorders
Uterine inflammation
|
5.0%
1/20 • Baseline to Week 96
|
|
Respiratory, thoracic and mediastinal disorders
Hyperventilation
|
5.0%
1/20 • Baseline to Week 96
|
|
Respiratory, thoracic and mediastinal disorders
Paranasal cyst
|
5.0%
1/20 • Baseline to Week 96
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
5.0%
1/20 • Baseline to Week 96
|
|
Respiratory, thoracic and mediastinal disorders
Upper-airway cough syndrome
|
5.0%
1/20 • Baseline to Week 96
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
5.0%
1/20 • Baseline to Week 96
|
|
Skin and subcutaneous tissue disorders
Alopecia areata
|
5.0%
1/20 • Baseline to Week 96
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
5.0%
1/20 • Baseline to Week 96
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
5.0%
1/20 • Baseline to Week 96
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
- Publication restrictions are in place
Restriction type: OTHER