ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 21 of 21 for:    pediatric multiple sclerosis

Phase 3 Efficacy and Safety Study of BG00012 in Pediatric Subjects With Relapsing-remitting Multiple Sclerosis (RRMS) (CONNECT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02283853
Recruitment Status : Recruiting
First Posted : November 5, 2014
Last Update Posted : May 25, 2018
Sponsor:
Information provided by (Responsible Party):
Biogen

Brief Summary:
The main objectives of Part 1 are as follows: To evaluate the safety, tolerability, and efficacy of BG00012 in pediatric subjects with RRMS, as compared with a disease-modifying treatment and 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. The secondary objective of Part 2 is to describe the long-term MS outcomes of BG00012 in subjects who completed Week 96 in Part 1 of Study 109MS306.

Condition or disease Intervention/treatment Phase
Relapsing-Remitting Multiple Sclerosis Drug: dimethyl fumarate Drug: Interferon β-1a Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 142 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-Label, Randomized, Multicenter, Multiple-Dose,Active-Controlled, Parallel-Group, Efficacy and Safety Study of BG00012 in Children From 10 to Less Than 18 Years of Age With Relapsing-Remitting Multiple Sclerosis, With Optional Open-Label Extension
Actual Study Start Date : August 28, 2014
Estimated Primary Completion Date : May 21, 2020
Estimated Study Completion Date : January 2, 2025

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: BG00012
Participants will receive 120 mg capsule(s) taken orally.
Drug: dimethyl fumarate
administered orally
Other Names:
  • BG00012
  • Tecfidera
Active Comparator: IFN β-1a (Avonex)
Participants will receive the recommended dose of 30 μg (weekly)
Drug: Interferon β-1a
administered by intramuscular injection
Other Name: Avonex



Primary Outcome Measures :
  1. Proportion of participants free of new/newly enlarging T2 hyperintense lesions on brain MRI scans [ Time Frame: At week 96 ]
    Part 1

  2. Number of participants that experience Adverse Events (AEs) or Serious Adverse Events (SAEs) [ Time Frame: Up to 5 years ]
    Part 2 will be an optional open-label extension phase in subjects who complete Part 1 and who meet the Part 2 entry criteria.

  3. Number of participants who discontinue study treatment due to an AE [ Time Frame: Up to 5 years ]
    Part 2


Secondary Outcome Measures :
  1. The number of new/newly enlarging T2 hyperintense lesions on brain MRI scans [ Time Frame: At Week 24 and Week 96 ]
  2. Proportion of participants free of new/newly enlarging T2 hyperintense lesions on brain MRI scans [ Time Frame: At Week 24 and Week 48 ]
  3. Proportion of participants free of new MRI activity as measured by brain MRI scans [ Time Frame: At Weeks 24, 48 and 96 ]
    New MRI Activitiy includes: Gd-enhancing MRI lesions on brain MRI scans; New T2 MRI lesions on brain MRI scans and newly enlarging MRI lesions on brain MRI scans

  4. Time to first relapse [ Time Frame: Up to Week 96 ]
  5. Proportion of participants who do not experience relapse [ Time Frame: Up to Week 96 ]
  6. Annualized relapse rate [ Time Frame: At Weeks 48 and 96 ]
  7. Number of participants that experience Adverse Events (AEs) and serious adverse events (SAEs) [ Time Frame: Up to Week 96 ]
    Including prospective and follow-up of flushing, nausea, abdominal pain and diarrhea

  8. Fatigue as measured by the Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue Scale scores [ Time Frame: Up to Week 96 ]
    Multidimensional Fatigue Scale scores - designed as a generic symptom-specific instrument to measure fatigue in patients with acute and chronic health conditions as well as healthy school and community populations.

  9. Quality of Life as measured by the PedsQL [ Time Frame: Up to Week 96 ]
  10. Change from baseline to Week 96 in the Expanded Disability Status Scale (EDSS) score [ Time Frame: Up to Week 96 ]
    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.

  11. Vital signs, electrocardiograms (ECGs) and changes in clinical laboratory data, including monitoring of liver function, renal function, hematologic, and coagulation parameters [ Time Frame: Up to Week 96 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   10 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 of the following 3 conditions: a) at least 1 relapse within the last 12 months prior to Day 1 with a prior brain MRI demonstrating lesions consistent with MS; b) at least 2 relapses within the last 24 months prior to Day 1, with a prior brain MRI demonstrating lesions consistent with MS; c) 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.

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, fumaric acid esters, or interferon beta-1a (IFN Beta-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; intravenous (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 not 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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02283853


Contacts
Contact: Biogen clinicaltrials@biogen.com

  Show 71 Study Locations
Sponsors and Collaborators
Biogen
Investigators
Study Director: Medical Director Biogen

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT02283853     History of Changes
Other Study ID Numbers: 109MS306
2013-002318-11 ( EudraCT Number )
First Posted: November 5, 2014    Key Record Dates
Last Update Posted: May 25, 2018
Last Verified: May 2018

Keywords provided by Biogen:
Pediatrics

Additional relevant MeSH terms:
Sclerosis
Multiple Sclerosis
Multiple Sclerosis, Relapsing-Remitting
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Interferons
Interferon-beta
Interferon beta-1a
Dimethyl Fumarate
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Dermatologic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic