Trial Outcomes & Findings for Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With RRMS (NCT NCT02410200)

NCT ID: NCT02410200

Last Updated: 2017-10-23

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 22 participants
• Primary outcome timeframe: Baseline Period (Week -8 to Day 0), On-Treatment Assessment Period (Week 16 to Week 24)
• Results posted on: 2017-10-23

Participant Flow

Participant milestones

Measure	BG00012 BG00012 taken orally at a dose of 120 mg twice daily (BID) for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Overall Study STARTED	22
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	BG00012 BG00012 taken orally at a dose of 120 mg twice daily (BID) for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Overall Study Adverse Event	2

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	BG00012 n=22 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Age, Continuous	15.8 years STANDARD_DEVIATION 1.18 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline Period (Week -8 to Day 0), On-Treatment Assessment Period (Week 16 to Week 24)

Population: Primary Analysis Population: participants having new or newly enlarging T2 lesions during the Baseline period with a post-baseline assessment.

Outcome measures

Measure	BG00012 n=15 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
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	-7.9 lesions Standard Deviation 16.23

SECONDARY outcome

Timeframe: Day 8

Population: All participants who received at least 1 dose of BG00012 and had at least 1 pharmacokinetic parameter available.

Outcome measures

Measure	BG00012 n=21 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Maximum Observed Plasma Concentration (Cmax)	1998.62 ng/mL Standard Deviation 1286.467

SECONDARY outcome

Timeframe: Day 8

Population: All participants who received at least 1 dose of BG00012 and had at least 1 pharmacokinetic parameter available.

Outcome measures

Measure	BG00012 n=21 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Time to Reach Maximum Observed Plasma Concentration (Tmax)	4.20 hours Standard Deviation 1.543

SECONDARY outcome

Timeframe: Day 8

Population: All participants who received at least 1 dose of BG00012 and had at least 1 pharmacokinetic parameter available.

Outcome measures

Measure	BG00012 n=17 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Apparent Clearance (CL/F)	74.45 L/h Standard Deviation 30.185

SECONDARY outcome

Timeframe: Day 8

Population: All participants who received at least 1 dose of BG00012 and had at least 1 pharmacokinetic parameter available.

Outcome measures

Measure	BG00012 n=14 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Apparent Volume of Distribution (V/F)	98.19 L Standard Deviation 91.679

SECONDARY outcome

Timeframe: Day 8

Population: All participants who received at least 1 dose of BG00012 and had at least 1 pharmacokinetic parameter available.

Outcome measures

Measure	BG00012 n=14 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Half-Life Lambda z	0.84 hours Standard Deviation 0.408

SECONDARY outcome

Timeframe: Day 8

Population: All participants who received at least 1 dose of BG00012 and had at least 1 pharmacokinetic parameter available.

Outcome measures

Measure	BG00012 n=14 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Area Under the Concentration-Time Curve From Time 0 to Infinity (AUC0-inf)	3630.52 h*mcg/mL Standard Deviation 1153.768

SECONDARY outcome

Timeframe: Up to Week 28

Population: All participants who received at least 1 dose of BG00012.

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.

Outcome measures

Measure	BG00012 n=22 Participants BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Any event	20 participants
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Moderate or severe event	7 participants
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Severe event	1 participants
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Event related to BG00012	16 participants
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Serious event	5 participants
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Serious event related to BG00012	0 participants
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Discontinued treatment due to an event	2 participants
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Withdrew from study due to an event	2 participants

Adverse Events

BG00012

Serious events: 5 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	BG00012 n=22 participants at risk BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Ear and labyrinth disorders Vertigo	4.5% 1/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Nervous system disorders Multiple sclerosis relapse	18.2% 4/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.

Other adverse events

Measure	BG00012 n=22 participants at risk BG00012 taken orally at a dose of 120 mg BID for the first 7 days and at a dose of 240 mg BID thereafter for 24 weeks.
Ear and labyrinth disorders Vertigo	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Gastrointestinal disorders Abdominal pain	27.3% 6/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Gastrointestinal disorders Abdominal pain upper	18.2% 4/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Gastrointestinal disorders Nausea	18.2% 4/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Gastrointestinal disorders Vomiting	13.6% 3/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
General disorders Fatigue	13.6% 3/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Infections and infestations Nasopharyngitis	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Infections and infestations Upper respiratory tract infection	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Infections and infestations Viral upper respiratory tract infection	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Investigations Lymphocyte count decreased	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Nervous system disorders Headache	18.2% 4/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Nervous system disorders Multiple sclerosis relapse	31.8% 7/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Respiratory, thoracic and mediastinal disorders Cough	13.6% 3/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Skin and subcutaneous tissue disorders Alopecia	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Vascular disorders Flushing	45.5% 10/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.
Reproductive system and breast disorders Dysmenorrhoea	9.1% 2/22 • From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up. Treatment-emergent events are presented.

Additional Information

Biogen Study Medical Director

Biogen

Email: clinicaltrials@biogen.com

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