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

NCT03958877 | Active Not Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: 105MS3062018-003008-382023-505624-56
• Study Type: interventional
• Target: 152
• Locations: 21 countries
• Sites: 62

Brief 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.

Conditions

Multiple Sclerosis, Relapsing-Remitting

Outcome Measures

Primary Outcomes (2)

• Part 1: Annualized Relapse Rate (ARR) at 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.

  Week 48

• Part 2: Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Treatment Discontinuation

  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.

  From Week 96 to Week 196

Secondary Outcomes (40)

• Part 1: ARR at Week 96

  Week 96

• Part 1: Percentage of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans at Weeks 24, 48, and 96

  Weeks 24, 48, and 96

• Part 1: Percentage of Participants Free of New MRI Activity in the Brain (Free of Gadolinium [Gd]-Enhancing Lesions and New or Newly Enlarging T2 Hyperintense Lesions) at Weeks 24, 48, and 96

  Weeks 24, 48, and 96

• Part 1: Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain MRI Scans at Weeks 24, 48, and 96

  Weeks 24, 48, and 96

• Part 1: Number of Gd-Enhancing Lesions on Brain MRI Scans at Weeks 24, 48, and 96

  Weeks 24, 48, and 96

Study Arms (2)

BIIB017 (peginterferon beta-1a)

EXPERIMENTAL

Participants will receive subcutaneous (SC) injection of BIIB017 (peginterferon beta-1a) 63 microgram (μg) on Day 1, followed by 94 μg at Week 2, followed by 125 μg at Week 4, and then 125 μg SC injection every 2 weeks up to Week 96 in Part 1 of the study. Eligible participants who enter optional Part 2 of the study will receive 125 μg SC injections of BIIB017 every 2 weeks for 96 Weeks.

Drug: BIIB017 (peginterferon beta-1a)

Avonex

ACTIVE COMPARATOR

Participants will receive Avonex (interferon beta type 1a) starting at a dose of 7.5 μg on Day 1, followed by an increase of 7.5 μg each week for 3 weeks, followed by 30 μg intramuscular (IM) injections every week up to Week 96 in Part 1 of the study. Eligible participants who enter optional Part 2 of the study will receive 125 μg SC injections of BIIB017 every 2 weeks for 96 Weeks.

Drug: Interferon beta type 1a

Interventions

BIIB017 (peginterferon beta-1a) DRUG

Administered as specified in the treatment arm

Also known as: PLEGRIDY

BIIB017 (peginterferon beta-1a)

Interferon beta type 1a DRUG

Administered as specified in the treatment arm

Also known as: Avonex

Avonex

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biogen: lead

Study Sites (65)

UC San Diego Health

La Jolla, California, 92024, United States

Location

UNC Hospitals

Chapel Hill, North Carolina, 27514, United States

Location

Meridian Clinical Research

Norfolk, Virginia, 23502, United States

Location

Hospital Italiano de Buenos Aires

Ciudad Autonoma Buenos Aires, Buenos Aires, 1199, Argentina

Location

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

Location

Universitair Ziekenhuis Ghent

Ghent, East Flanders, 9000, Belgium

Location

Clinique CHC MontLégia

Liège, Wallonia, 4000, Belgium

Location

MHATNP 'Sv.Naum', EAD

Sofia, 1113, Bulgaria

Location

University Hospital Centre Split

Split, Dalmatia, 21000, Croatia

Location

Children's Hospital Zagreb

Zagreb, 10000, Croatia

Location

Clinical Hospital Center 'Sestre Milosrdnice'

Zagreb, 10000, Croatia

Location

University Hospital Centre Zagreb

Zagreb, 10000, Croatia

Location

Fakultni nemocnice Hradec Kralove

Hradec Králové, 50333, Czechia

Location

CHU Strasbourg - Hôpital Hautepierre

Strasbourg, Bas Rhin, 67098, France

Location

Hopital Purpan

Toulouse, Haute Garonne, 31059, France

Location

Hopital Gui de Chauliac

Montpellier, Herault, 34295, France

Location

Hopital Roger Salengro - CHU Lille

Lille, Nord, 59037, France

Location

Hôpital Bicêtre

Le Kremlin-Bicêtre, Val De Marne, 94275, France

Location

Universitaetsklinikum Freiburg

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

Universitaetsmedizin Goettingen

Göttingen, Lower Saxony, 37075, Germany

Location

St. Josef-Hospital Universitaetsklinikum

Bochum, North Rhine-Westphalia, 44791, Germany

Location

Charité - Campus Virchow-Klinikum

Berlin, 13353, Germany

Location

General Hospital of Larissa

Larissa, Thessaly, 41110, Greece

Location

General Hospital of Thessaloniki 'Hippokration'

Thessaloniki, 54642, Greece

Location

Pecsi Tudomanyegyetem KK

Pécs, Baranya, 7623, Hungary

Location

Debreceni Egyetem

Debrecen, Hajdú-Bihar, 4032, Hungary

Location

Semmelweis Egyetem

Budapest, 1083, Hungary

Location

Hadassah University Hospital - Ein Kerem

Jerusalem, Levant, 9112001, Israel

Location

Schneider Children's Medical Center

Petach-Tikva, Tel Aviv, 4920235, Israel

Location

Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

Florence, 50139, Italy

Location

Azienda Ospedaliera Universitaria 'Federico II'

Napoli, 80131, Italy

Location

Azienda Ospedaliera Universitaria- Università degli Studi della Campania "Luigi Vanvitelli"

Napoli, 80138, Italy

Location

Ibn Sina Hospital

Ash Shuwaykh, Shuwaikh, 12345, Kuwait

Location

Centro Hospitalar e Universitário Lisboa Norte E.P.E.

Lisbon, Lisbon District, 1649-035, Portugal

Location

Hospital Beatriz Ângelo

Loures, Lisbon District, 2674-514, Portugal

Location

Hospital de Braga

Braga, Minho, 4710-243, Portugal

Location

Centro Hospitalar e Universitário de Coimbra E.P.E. - Hospital Pediátrico

Coimbra, 3000-602, Portugal

Location

Centro Hospitalar do Porto, E.P.E. - Hospital de Santo António

Porto, 4099-001, Portugal

Location

SBEI HPE 'Bashkir State Medical University' of the MoH of the RF

Ufa, Bashkortostan Republic, 450077, Russia

Location

FSBI "Federal Siberian Scientific-Clinical Center of FMBA"

Krasnoyarsk, Krasnoyarsk Krai, 660037, Russia

Location

Nebbiolo LLC

Tomsk, Oblast, 634009, Russia

Location

LLC National center for socially significan disease

Saint Petersburg, Sankt-Peterburg, 197110, Russia

Location

SAIH 'Kemerovo Regional Clinical Hospital'

Kemerovo, Siberia, 650066, Russia

Location

RSBIH 'Belgorod Regional Clinical Hospital of Saint Ioasaf'

Belgorod, 308007, Russia

Location

SBHI

Moscow, 119602, Russia

Location

SBIH of Moscow region "Moscow Regional Scientific & Research

Moscow, 129110, Russia

Location

SBEI HPE 'Rostov State Medical University' of the MoH of the RF

Rostov-on-Don, 344022, Russia

Location

State Budgetary Institution of Healthcare of Yaroslavl region 'Clinical Hospital # 2'

Yaroslavl, 150000, Russia

Location

King Faisal Specialist Hospital & Research Center

Jeddah, Mecca Region, 40047, Saudi Arabia

Location

King Saud University

Riyadh, 11461, Saudi Arabia

Location

Clinic of Neurology and Psychiatry for Children and Youth

Belgrade, Balkans, 11000, Serbia

Location

University Children Hospital

Belgrade, Balkans, 11000, Serbia

Location

Mother and Child Health Care Institute of Serbia ,,Dr Vukan Cupic''

Belgrade, Balkans, 11070, Serbia

Location

Narodny ustav detskych chorob

Bratislava, 83340, Slovakia

Location

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

Hospital Universitario Virgen de la Arrixaca

El Palmar, Murcia, 30120, Spain

Location

Hospital Universitario Reina Sofia

Córdoba, 14011, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hopital Razi

Manouba, Mannouba, 2010, Tunisia

Location

Hôpital Fattouma Bourghiba

Monastir, 5000, Tunisia

Location

Hôpital Habib Bourguiba

Sfax, 3029, Tunisia

Location

Gazi University Medical Faculty Clinical Research Unit

Ankara, 06500, Turkey (Türkiye)

Location

Akdeniz University Medical Faculty

Antalya, 07059, Turkey (Türkiye)

Location

Izmir Dr. Behcet Uz Cocuk Hastaliklari ve Cerrahisi Egitim ve Arastirma Hastanesi

Izmir, 35210, Turkey (Türkiye)

Location

Ondokuz Mayis Univ. Med. Fac.

Samsun, 55139, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Multiple Sclerosis, Relapsing-Remitting

Interventions

peginterferon beta-1a; Interferon beta-1a

Condition Hierarchy (Ancestors)

Multiple Sclerosis; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Interferon-beta; Interferon Type I; Interferons; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2019
• First Posted: May 22, 2019
• Study Start: October 18, 2019
• Primary Completion (Estimated): May 20, 2027
• Study Completion (Estimated): May 20, 2027
• Last Updated: September 11, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

CZ (1); TU (4); CR (4); SA (2); RU (10); KU (1); IL (2); US (3); BE (2); IT (3); AR (1); GR (2); SE (3); FR (5); AU (1); ES (4); HU (3); DE (4); PT (5); BU (1); SL (1)