Relapsing-Remitting Multiple Sclerosis MedDRA version: 20.0 Level: SOC Classification code 10029205 Term: Nervous system disorders System Organ Class: 10029205 - Nervous system disorders MedDRA version: 21.1 Level: PT Classification code 10063399 Term: Relapsing-remitting multiple sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Biogen Idec Research Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: key inclusion criteria: - Must have a body weight of =30 kg. - Must have a diagnosis of RRMS (consensus definition for pediatric RRMS [Krupp 2013]). - Must be ambulatory with a baseline EDSS score between 0 and 5.5, inclusive. - Must have experienced at least 1 relapse within the last 12 months prior to Day 1, or at least 2 relapses within the last 24 months prior to Day 1, with a prior brain MRI demonstrating lesions consistent with MS, or evidence of Gd enhancing lesions of the brain on an MRI performed within the 6 weeks prior to Day 1. - Must be neurologically stable, with no evidence of relapse within 50 days prior to Day 1 and no evidence of corticosteroid treatment within 30 days prior to Day 1. - Subjects of childbearing potential who are sexually active must be willing to practice effective contraception during the study and be willing and able to continue contraception for at least 30 days after their final dose of study treatment. Part 2: - Subjects who have completed Week 96 in Part 1 NOTE: Other Protocol defined inclusion criteria may apply Are the trial subjects under 18? yes Number of subjects for this age range: 132 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Key exclusion criteria: - Primary progressive, secondary progressive, or progressive relapsing MS (as defined by [Lublin and Reingold 1996]). These conditions require the presence of continuous clinical disease worsening over a period of at least 3 months. Subjects with these conditions may also have superimposed relapses but are distinguished from relapsing remitting subjects by the lack of clinically stable periods or clinical improvement. - Disorders mimicking MS, such as other demyelinating disorders (e.g., acute disseminated encephalomyelitis), systemic autoimmune disorders (e.g., Sjögren disease, lupus erythematosus), metabolic disorders (e.g., dystrophies), and infectious disorders. - History of premalignant or malignant disease. Subjects with basal cell carcinoma that has been completely excised prior to screening will remain eligible. - History of severe allergic, anaphylactic reactions, or known drug hypersensitivity to DMF or fumaric acid esters or interferon ß-1a (IFN ß- 1a). - History of abnormal laboratory results indicative of any significant endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, renal, and/or any other major disease that would preclude participation in a clinical study. - History of clinically significant cardiovascular, pulmonary, GI, dermatologic, growth, developmental, psychiatric (including depression), neurologic (other than MS), and/or other major disease that would preclude participation in a clinical study. - History of human immunodeficiency virus. - An MS relapse that has occurred within 50 days prior to Day 1 AND/OR the subject has not stabilized from a previous relapse prior to Day 1. - Other unspecified reasons that, in the opinion of the Investigator or Biogen Idec, make the subject unsuitable for enrollment. - For the PD/PK subset of subjects: subjects unable to swallow the BG00012 capsule whole. Key Treatment history - Any previous treatment with Fumaderm (fumaric acid esters) or BG00012. - Prior treatment with any of the following: total lymphoid irradiation, cladribine, T-cell or T-cell receptor vaccination, any therapeutic monoclonal antibody, with the exception of rituximab or natalizumab - Prior treatment with any of the following medications within the 12 months prior to Day 1: mitoxantrone, cyclophosphamide, rituximab - Prior treatment with any of the following medications or procedures within 6 months prior to Day 1: fingolimod, teriflunomide, natalizumab, cyclosporine, azathioprine, methotrexate, mycophenolate mofetil, laquinimod, IV immunoglobulin, plasmapheresis or cytapheresis - Treatment with any of the following medications within 30 days prior to Day 1: - steroids (IV or oral corticosteroid treatment, including agents that may act through the corticosteroid pathway [e.g., low dose naltrexone]), 4- aminopyridine or related products (except subjects on a stable dose of controlled-release fampridine for 3 months) Part 2; - Any significant changes in medical history occurring after enrollment in Part 1 - Subjects who could not tolerate BG00012 in Part 1 NOTE: Other protocol-defined inclusion/exclusion criteria may apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objectives of part 1 are as follows: • To evaluate the safety, tolerability, and efficacy on the disease course of BG00012 (dimethyl fumarate) in pediatric participants with RRMS, as compared with a disease modifying treatment. • To assess pharmacokinetic (PK) and pharmacodynamic (PD) parameters in a representative subset of pediatric participants • To assess health outcomes and evolution of disability The primary objective of Part 2 is to evaluate the long-term safety of BG00012 in subjects who completed week 96 in Part 1 of Study 109MS306.;Secondary Objective: Not Applicable;Primary end point(s): The primary endpoint of part 1 of the study is the proportion of participants free of new/newly enlarging T2 hyperintense lesions on brain MRI scans at Week 96. The primary endpoint of the Part 2 is the incidence of adverse events, serious adverse events and discontinuations of BG00012 due to an adverse event;Timepoint(s) of evaluation of this end point: Part 1 - Day 1 (baseline visit), weeks 24, 48, 72, 96 Part 2 - up to week 244 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints of part 1 are as follows: • Number of new/newly enlarging T2 hyperintense lesions on brain MRI scans at Weeks 24 and 96 • Proportion of participants free of new/newly enlarging T2 hyperintense lesions on brain MRI scans at Weeks 24 and 48 • Proportion of participants free of new MRI activity as measured by brain MRI scans (new MRI activity includes: Gd enhancing MRI lesion on brain MRI scans; new T2 MRI lesions on brain MRI scans and newly enlarging LRI lesions on brain MRI scans) at Weeks 24, 48, and 96 • Time to first relapse up to week 96 • Proportion of participants who do not experience relapse up to Week 96 • Annualized relapse rate at Weeks 48 and 96 • Number of participants that experience adverse events (AEs) and serious adverse events (SAEs), including prospective and follow up of flushing, nausea, abdominal pain, and diarrhea up to week 96 • Fatigue as measured by the Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue Scale scores (Multidimensional Fatigue Scale scores designed as a generic symptomspecific instrument to measure fatigue in patients with acute and chronic health conditions as well as healthy school and community populations.) up to week 96 • Quality of Life as measured by the PedsQL up to week 96 • Change from baseline to Week 96 in the Expanded Disability Status Scale (EDSS score; The EDSS measures the disability status of people with multiple sclerosis on a scale that ranges from 0 to 10. The first levels 1.0 to 4.5 refer to people with a high degree of ambulatory ability and the subsequent levels 5.0 to 9.5 refer to the loss of ambulatory ability. The range of main categories include (0) = normal neurologic exam; to (5) = ambulatory without aid or rest for 200 meters; disability severe enough to impair full daily activities; to (10) = death due to MS.) up to week 96 • Vital signs, electrocardiograms (ECGs) and changes in clinical laboratory | — |
Countries
Belgium, Bulgaria, Canada, Czechia, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, Kuwait, Poland, Serbia, Spain, Sweden, Turkey, United Kingdom, United States
Contacts
Public ContactMedical Director
Biogen Idec Research Limited
Outcome results
None listed