A Study to Evaluate the Safety, Tolerability, and Efficacy of BIIB017 (Peginterferon Beta-1a) in Pediatric Participants for the Treatment of Relapsing-Remitting Multiple Sclerosis
BIIB017 (peginterferon beta-1a): Administered as specified in the treatment arm
Interferon beta type 1a: Administered as specified in the treatment arm
Study summary
This study will evaluate the safety, tolerability, and descriptive efficacy of BIIB017 in pediatric participants with relapsing-remitting multiple sclerosis (RRMS) and to assess the pharmacokinetics (PK) of BIIB017 in pediatric participants with RRMS in Part 1. In Part 2, the study will evaluate the long-term safety of BIIB017 and further describe safety and the long-term multiple sclerosis (MS) outcomes after BIIB017 treatment in participants who completed the study treatment at Week 96 in Part 1 of the study.
Eligibility
Sex
ALL
Min age
10 Years
Max age
18 Years
Healthy volunteers
No
Key Inclusion Criteria:
Part 1:
* Must have a diagnosis of RRMS as defined by the revised consensus definition for pediatric MS.
* Must have an EDSS score between 0.0 and 5.5.
* Must have experienced \>= 1 relapse in the 12 months prior to randomization (Day 1) or \>= 2 relapses in the 24 months prior to randomization (Day 1) or have evidence of asymptomatic disease activity (Gd-enhancing lesions) on brain MRI in the 6 months prior to randomization (Day 1).
Part 2:
• Participants who completed the study treatment in Part 1 (Week 96 Visit), as per protocol.
Key Exclusion Criteria:
Part 1:
* Primary progressive, secondary progressive, or progressive relapsing MS. These conditions require the presence of continuous clinical disease worsening over a period of at least 3 months. Participants with these conditions may also have superimposed relapses but are distinguished from relapsing participants by the lack of clinically stable periods or clinical improvement.
* History of severe allergic or anaphylactic reactions or known drug hypersensitivity.
* Known allergy to any component of Avonex or BIIB017 formulation.
* Occurrence of an MS relapse that has occurred within 30 days prior to randomization (Day 1) and/or the participant has not stabilized from a previous relapse prior to randomization (Day 1).
* Any previous treatment with PEGylated human IFN β-1a.
Part 2:
* Any significant changes in medical history occurring after enrollment in Part 1, including laboratory test abnormalities or current clinically significant conditions that, in the opinion of the Investigator, would have excluded the participant's participation in Part 1. The Investigator must re-assess the participant's medical fitness for participation and consider any factors that would preclude treatment.
* The participant could not tolerate BIIB017 in Part 1.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Primary outcome measure(s)
Part 1: Annualized Relapse Rate (ARR) at Week 48 — Week 48 A multiple sclerosis (MS) relapse is defined as the onset of new or recurrent neurologic symptoms not associated with fever or infection, lasting at least 24 hours, and accompanied by new objective neurological findings. ARR is calculated as the total number of relapses in each treatment group adjusted for the duration of study treatment in person-years.
Part 2: Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Treatment Discontinuation — From Week 96 to Week 196 An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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