Clinical Trial Results:
Open-Label, Multicenter, Multiple-Dose Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With Relapsing-Remitting Multiple Sclerosis Aged 10 to 17 Years
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Summary
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EudraCT number |
2014-005003-24 |
Trial protocol |
LV DE CZ BG PL BE |
Global end of trial date |
23 Sep 2016
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Results information
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Results version number |
v2(current) |
This version publication date |
14 Oct 2017
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First version publication date |
05 Apr 2017
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Other versions |
v1 |
Version creation reason |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
109MS202
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02410200 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Biogen
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Sponsor organisation address |
225 Binney Street, Cambridge, Massachusetts, United States, 02142
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Public contact |
Biogen Study Medical Director, Biogen, clinicaltrials@biogen.com
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Scientific contact |
Biogen Study Medical Director, Biogen, clinicaltrials@biogen.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
23 Sep 2016
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
23 Sep 2016
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The primary objective of this study is to evaluate the effect of BG00012 (dimethyl fumarate) on brain magnetic resonance imaging (MRI) lesions in pediatric participants with relapsing-remitting multiple sclerosis (RRMS). The secondary objectives of this study are to characterize the pharmacokinetics of BG00012 in pediatric participants with RRMS and to evaluate the safety and tolerability of BG00012 in pediatric participants with RRMS.
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Protection of trial subjects |
Written informed consent was obtained from each subject prior to evaluations being performed for eligibility. Subjects were given adequate time to review the information in the informed consent and were allowed to ask, and have answered, questions concerning all portions of the conduct of the study. Through the informed consent process each subject was made aware of the purpose of the study, the procedures, the benefits and risks of the study, the discomforts and the precautions taken. Any side effects or other health issues occurring during the study were followed up by the study doctor. Subjects were able to stop taking part in the study at any time without giving any reason.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
07 Sep 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Poland: 5
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Country: Number of subjects enrolled |
Bulgaria: 3
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Country: Number of subjects enrolled |
Germany: 3
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Country: Number of subjects enrolled |
Kuwait: 3
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Country: Number of subjects enrolled |
Lebanon: 2
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Country: Number of subjects enrolled |
Turkey: 2
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Country: Number of subjects enrolled |
Belgium: 1
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Country: Number of subjects enrolled |
Czech Republic: 1
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Country: Number of subjects enrolled |
Latvia: 1
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Country: Number of subjects enrolled |
United States: 1
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Worldwide total number of subjects |
22
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EEA total number of subjects |
14
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
22
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||
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Pre-assignment
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Screening details |
Subject eligibility for the study was determined within 4 weeks prior to the Baseline Period. | ||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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BG00012 | ||||||||||
Arm description |
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. | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
BG00012
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Investigational medicinal product code |
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Other name |
dimethyl fumarate DMF
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Pharmaceutical forms |
Gastro-resistant capsule, hard
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Routes of administration |
Oral use
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Dosage and administration details |
Directions for Handling and Administration were followed.
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Baseline characteristics reporting groups
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Reporting group title |
BG00012
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Reporting group description |
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. | ||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
BG00012
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Reporting group description |
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. | ||
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End point title |
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 [1] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Baseline Period (Week -8 to Day 0), On-Treatment Assessment Period (Week 16 to Week 24)
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Statistical analysis is provided in V1 document attached to this endpoint presentation. (Analysis could not be entered into EudraCT due to system limitations.) |
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Attachments |
Untitled (Filename: Statistical Analysis for Primary Endpoint.docx) |
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End point title |
Maximum Observed Plasma Concentration (Cmax) | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Day 8
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| No statistical analyses for this end point | |||||||||
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End point title |
Time to Reach Maximum Observed Plasma Concentration (Tmax) | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Day 8
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| No statistical analyses for this end point | |||||||||
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End point title |
Apparent Clearance (CL/F) | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Day 8
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| No statistical analyses for this end point | |||||||||
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End point title |
Apparent Volume of Distribution (V/F) | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Day 8
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| No statistical analyses for this end point | |||||||||
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End point title |
Half-Life Lambda z | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Day 8
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| No statistical analyses for this end point | |||||||||
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End point title |
Area Under the Concentration-Time Curve from Time 0 to Infinity (AUC0-inf) | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Day 8
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) and Serious Adverse events (SAEs) | ||||||||||||||||||||||
End point description |
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.
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End point type |
Secondary
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End point timeframe |
Up to Week 28
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| No statistical analyses for this end point | |||||||||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
From first dose of study drug through end of treatment period (Week 24 ±7 days) plus 4 weeks follow-up.
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Adverse event reporting additional description |
Treatment-emergent events are presented.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
19
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Reporting groups
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Reporting group title |
BG00012
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Reporting group description |
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. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
