Phase 3 Efficacy and Safety Study of BG00012 in Pediatric Subjects With relapsing-remitting multiple sclerosis (RRMS).
- Conditions
- Relapsing-Remitting Multiple SclerosisMedDRA version: 18.1Level: SOCClassification code 10029205Term: Nervous system disordersSystem Organ Class: 10029205 - Nervous system disordersMedDRA version: 18.1Level: PTClassification code 10063399Term: Relapsing-remitting multiple sclerosisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2013-002318-11-ES
- Lead Sponsor
- Biogen Idec Research Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 142
key inclusion criteria:
- Must have a body weight of ?30 kg.
- Must have a diagnosis of RRMS (consensus definition for pediatric RRMS [Krupp 2007]).
- 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 show 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 142
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
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 or anaphylactic reactions, or known drug hypersensitivity to DMF or fumaric acid esters.
- 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
- Prior treatment with any of the following within 3 months prior to Day 1: glatiramer acetate, interferon-alpha, interferon ? (subjects who are positive for neutralizing antibodies to interferon-? may receive interferon-? treatment up to 2 weeks prior to Day 1)
- 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)
NOTE: Other protocol-defined inclusion/exclusion criteria may apply
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The main objectives of the study are as follows:<br>? To evaluate the safety, tolerability, and effect on the disease course of BG00012 (dimethyl fumarate) in pediatric participants with RRMS, as compared with a disease modifying treatment.<br>? To assess pharmacokinetic (PK) and pharmacodynamic (PD) parameters in a representative subset of pediatric participants<br>? To assess health outcomes and evolution of disability;Secondary Objective: Not Applicable;Primary end point(s): The primary endpoint of the study is the proportion of participants free of new/newly enlarging T2 hyperintense lesions on brain MRI scans at Week 96.;Timepoint(s) of evaluation of this end point: Day 1 (baseline visit), weeks 24, 48, 72, 96
- Secondary Outcome Measures
Name Time Method