Trial Outcomes & Findings for BG00012 and Delay of Disability Progression in Secondary Progressive Multiple Sclerosis (NCT NCT02430532)
NCT ID: NCT02430532
Last Updated: 2017-04-26
Results Overview
Time to onset of confirmed progression of disability is defined as 1 or more of the following criteria, confirmed at ≥ 6 months after start of treatment and at Week 108 using 1 or more of the following assessments: Expanded Disability Status Scale (EDSS) score increased from Baseline of ≥ 1 point if baseline EDSS ≤ 5.5, or ≥ 0.5 point if Baseline EDSS ≥ 6.0; Timed 25-Foot Walk (T25FW) ≥ 20% increase from Baseline in the time taken for the 25-foot walk; worsening on the 9-Hole Peg Test (9HPT; ≥ 20% increase from Baseline in the time taken for the 9HPT, confirmed in the same hand). The EDSS measures disability status on a scale ranging from 0 to 10, with higher scores indicating more disability. The T25FW is a quantitative mobility and leg function performance test where the participant is timed while walking for 25 feet. The 9HPT is a quantitative test of upper extremity function that measures the time it takes to place 9 pegs into 9 holes and then remove the pegs.
TERMINATED
PHASE3
58 participants
Up to 108 weeks
2017-04-26
Participant Flow
Participant milestones
| Measure |
Placebo
BG00012 120 mg capsule orally once a day (QD) supplemented with matching placebo capsules for the first 4 weeks of treatment. Matched placebo capsules only thereafter for up to 108 weeks.
|
Tecfidera 240 mg BID
BG00012 120 mg orally twice daily (BID) for 1 week, followed by BG00012 240 mg orally BID beginning on Day 8 for up to 108 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
30
|
28
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
30
|
28
|
Reasons for withdrawal
| Measure |
Placebo
BG00012 120 mg capsule orally once a day (QD) supplemented with matching placebo capsules for the first 4 weeks of treatment. Matched placebo capsules only thereafter for up to 108 weeks.
|
Tecfidera 240 mg BID
BG00012 120 mg orally twice daily (BID) for 1 week, followed by BG00012 240 mg orally BID beginning on Day 8 for up to 108 weeks.
|
|---|---|---|
|
Overall Study
Sponsor Decision to Stop Study Early
|
30
|
25
|
|
Overall Study
Consent Withdrawn
|
0
|
2
|
|
Overall Study
Adverse Event
|
0
|
1
|
Baseline Characteristics
BG00012 and Delay of Disability Progression in Secondary Progressive Multiple Sclerosis
Baseline characteristics by cohort
| Measure |
Placebo
n=30 Participants
BG00012 120 mg capsule orally QD supplemented with matching placebo capsules for the first 4 weeks of treatment. Matched placebo capsules only thereafter for up to 108 weeks.
|
Tecfidera 240 mg BID
n=28 Participants
BG00012 120 mg orally BID for 1 week, followed by BG00012 240 mg orally BID beginning on Day 8 for up to 108 weeks.
|
Total
n=58 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
50.7 years
STANDARD_DEVIATION 6.67 • n=5 Participants
|
49.6 years
STANDARD_DEVIATION 8.05 • n=7 Participants
|
50.2 years
STANDARD_DEVIATION 7.33 • n=5 Participants
|
|
Age, Customized
< 20 years
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Age, Customized
20 to 29 years
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Age, Customized
30 to 39 years
|
1 participants
n=5 Participants
|
4 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Age, Customized
40 to 49 years
|
10 participants
n=5 Participants
|
8 participants
n=7 Participants
|
18 participants
n=5 Participants
|
|
Age, Customized
>= 50 years
|
19 participants
n=5 Participants
|
16 participants
n=7 Participants
|
35 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 108 weeksPopulation: The limited number of participants enrolled and the early termination of the study resulted in efficacy data not collected, and efficacy outcomes not analyzed, as per the pre-specified plan of analysis.
Time to onset of confirmed progression of disability is defined as 1 or more of the following criteria, confirmed at ≥ 6 months after start of treatment and at Week 108 using 1 or more of the following assessments: Expanded Disability Status Scale (EDSS) score increased from Baseline of ≥ 1 point if baseline EDSS ≤ 5.5, or ≥ 0.5 point if Baseline EDSS ≥ 6.0; Timed 25-Foot Walk (T25FW) ≥ 20% increase from Baseline in the time taken for the 25-foot walk; worsening on the 9-Hole Peg Test (9HPT; ≥ 20% increase from Baseline in the time taken for the 9HPT, confirmed in the same hand). The EDSS measures disability status on a scale ranging from 0 to 10, with higher scores indicating more disability. The T25FW is a quantitative mobility and leg function performance test where the participant is timed while walking for 25 feet. The 9HPT is a quantitative test of upper extremity function that measures the time it takes to place 9 pegs into 9 holes and then remove the pegs.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 2 yearsPopulation: The limited number of participants enrolled and the early termination of the study resulted in efficacy data not collected, and efficacy outcomes not analyzed, as per the pre-specified plan of analysis.
MSWS-12 is a participant self-assessment of walking limitations due to multiple sclerosis (MS) during the past 2 weeks. It contains 12 items that measure the impact of MS on walking. Items are summed to generate a total score and transformed to a scale with a range of 0 to 100, where high scores indicate greater negative impact on walking.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 108Population: The limited number of participants enrolled and the early termination of the study resulted in efficacy data not collected, and efficacy outcomes not analyzed, as per the pre-specified plan of analysis.
The ABILHAND Questionnaire measures the participant's perceived difficulty in performing everyday manual activities in the last 3 months. Participants fill in the 56-item questionnaire by estimating their own difficulty or ease in performing each of the 56 activities. Items are summed to generate a total score and transformed to a scale with a range of 0 to 100, where high scores indicate greater impact on manual ability.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 108Population: The limited number of participants enrolled and the early termination of the study resulted in efficacy data not collected, and efficacy outcomes not analyzed, as per the pre-specified plan of analysis.
Whole brain volume is measured by magnetic resonance imaging (MRI).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 108Population: The limited number of participants enrolled and the early termination of the study resulted in efficacy data not collected, and efficacy outcomes not analyzed, as per the pre-specified plan of analysis.
The SDMT measures the time to pair abstract geometric symbols with specific numbers. The score is the number of correctly coded items from 0-110 in 90 seconds. A higher score indicates a better outcome.
Outcome measures
Outcome data not reported
Adverse Events
Placebo
Tecfidera 240 mg BID
Serious adverse events
| Measure |
Placebo
n=30 participants at risk
BG00012 120 mg capsule orally QD supplemented with matching placebo capsules for the first 4 weeks of treatment. Matched placebo capsules only thereafter for up to 108 weeks.
|
Tecfidera 240 mg BID
n=28 participants at risk
BG00012 120 mg orally BID for 1 week, followed by BG00012 240 mg orally BID beginning on Day 8 for up to 108 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Enteritis
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Nervous system disorders
Multiple sclerosis relapse
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Nervous system disorders
Trigeminal neuralgia
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Renal and urinary disorders
Calculus ureteric
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Renal and urinary disorders
Renal colic
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
3.6%
1/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
Other adverse events
| Measure |
Placebo
n=30 participants at risk
BG00012 120 mg capsule orally QD supplemented with matching placebo capsules for the first 4 weeks of treatment. Matched placebo capsules only thereafter for up to 108 weeks.
|
Tecfidera 240 mg BID
n=28 participants at risk
BG00012 120 mg orally BID for 1 week, followed by BG00012 240 mg orally BID beginning on Day 8 for up to 108 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
7.1%
2/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
7.1%
2/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
7.1%
2/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Infections and infestations
Upper respiratory tract infection
|
6.7%
2/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
0.00%
0/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Infections and infestations
Urinary tract infection
|
10.0%
3/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
7.1%
2/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Nervous system disorders
Multiple sclerosis relapse
|
0.00%
0/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
7.1%
2/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
|
Vascular disorders
Flushing
|
16.7%
5/30 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
17.9%
5/28 • Up to approximately 24 weeks (overall mean time on study of 14.34, with an overall mean time on study treatment of 9.58 weeks).
Data summaries of adverse events are descriptive in nature and the comparison between treatment groups might not be appropriate due to the small number of participants and limited study follow-up duration.
|
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