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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

Phase 1
Conditions
Relapsing Remitting Multiple Sclerosis (RRMS)
MedDRA version: 20.0Level: SOCClassification code 10029205Term: Nervous system disordersSystem Organ Class: 10029205 - Nervous system disorders
MedDRA version: 21.1Level: PTClassification code 10063399Term: Relapsing-remitting multiple sclerosisSystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Registration Number
EUCTR2018-003008-38-SK
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
Inclusion Criteria

Part 1
1. Must have a diagnosis of RRMS as defined by the revised consensus definition for
pediatric MS [Krupp 2013; Polman 2011].
2. Must have an EDSS score between 0.0 and 5.5.
3. 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
Subjects who completed the study treatment in Part 1 (Week 96 Visit), as per protocol.
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

Exclusion Criteria

PART 1
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. Subjects with these conditions may also have superimposed relapses but are distinguished from relapsing subjects by the lack of clinically stable periods or clinical improvement.
2. Occurrence of an MS relapse that has occurred within 30 days prior to randomization (Day 1) and/or the subject has not stabilized from a previous relapse prior to randomization (Day 1).
3. History of severe allergic or anaphylactic reactions or known drug hypersensitivity.
4. Known allergy to any component of Avonex or BIIB017 formulation.
5. Any previous treatment with PEGylated human IFN ß-1a.

PART 2
1. 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 subject’s participation in Part 1.
The Investigator must re-assess the subject’s medical fitness for participation and consider any factors that would preclude treatment.
2. The subject could not tolerate BIIB017 in Part 1.

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
NameTimeMethod
Main Objective: This study will evaluate the safety, tolerability, and descriptive efficacy of BIIB017 in pediatric participants with relapsing-remitting multiple sclerosis <br>(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 <br>safety of BIIB017 and further describe safety and the long-term multiple sclerosis (MS) outcomes after BIIB017 treatment in participants who completed <br>the study treatment at Week 96 in Part 1 of the study.;Secondary Objective: ;Primary end point(s): #1: Part 1: the annualized relapse rate (ARR) at Week 48.<br>#2: Part 2: Percentage of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs leading to Study Treatment Discontinuation.;Timepoint(s) of evaluation of this end point: #1: week 48<br>#2: Week 96 to Week 196
Secondary Outcome Measures
NameTimeMethod
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