Multiple Sclerosis, Relapsing-RemittingPhase 3NCT03958877
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
A Phase 3 study testing BIIB017 (peginterferon beta-1a) and Interferon beta type 1a in people with Multiple Sclerosis, Relapsing-Remitting, run by Biogen at 65 sites in 22 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 to 18 years
- Conditions the study is for
- Multiple Sclerosis, Relapsing-Remitting
- What is being tested?
- BIIB017 (peginterferon beta-1a) and Interferon beta type 1a
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 152 participants
- Where
- 65 sitesin 22 countries
- Study dates
- 18 Oct 2019to 20 May 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
65 sites in 22 countries.
65 sites in 22 countries
Russia10 sites
RSBIH 'Belgorod Regional Clinical Hospital of Saint Ioasaf'
Belgorod, 308007
SAIH 'Kemerovo Regional Clinical Hospital'
Kemerovo, Siberia, 650066
FSBI "Federal Siberian Scientific-Clinical Center of FMBA"
Krasnoyarsk, Krasnoyarsk Krai, 660037
SBHI
Moscow, 119602
SBIH of Moscow region "Moscow Regional Scientific & Research
Moscow, 129110
SBEI HPE 'Rostov State Medical University' of the MoH of the RF
Rostov-on-Don, 344022
LLC National center for socially significan disease
Saint Petersburg, Sankt-Peterburg, 197110
Nebbiolo LLC
Tomsk, Oblast, 634009
SBEI HPE 'Bashkir State Medical University' of the MoH of the RF
Ufa, Bashkortostan Republic, 450077
State Budgetary Institution of Healthcare of Yaroslavl region 'Clinical Hospital # 2'
Yaroslavl, 150000
France5 sites
Hôpital Bicêtre
Le Kremlin-Bicêtre, Val De Marne, 94275
Hopital Roger Salengro - CHU Lille
Lille, Nord, 59037
Hopital Gui de Chauliac
Montpellier, Herault, 34295
CHU Strasbourg - Hôpital Hautepierre
Strasbourg, Bas Rhin, 67098
Hopital Purpan
Toulouse, Haute Garonne, 31059
Portugal5 sites
Hospital de Braga
Braga, Minho, 4710-243
Centro Hospitalar e Universitário de Coimbra E.P.E. - Hospital Pediátrico
Coimbra, 3000-602
Centro Hospitalar e Universitário Lisboa Norte E.P.E.
Lisbon, Lisbon District, 1649-035
Hospital Beatriz Ângelo
Loures, Lisbon District, 2674-514
Centro Hospitalar do Porto, E.P.E. - Hospital de Santo António
Porto, 4099-001
Croatia4 sites
University Hospital Centre Split
Split, Dalmatia, 21000
Children's Hospital Zagreb
Zagreb, 10000
Clinical Hospital Center 'Sestre Milosrdnice'
Zagreb, 10000
University Hospital Centre Zagreb
Zagreb, 10000
Germany4 sites
Charité - Campus Virchow-Klinikum
Berlin, 13353
St. Josef-Hospital Universitaetsklinikum
Bochum, North Rhine-Westphalia, 44791
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106
Universitaetsmedizin Goettingen
Göttingen, Lower Saxony, 37075
Spain4 sites
Hospital Universitario Reina Sofia
Córdoba, 14011
Hospital Universitario Virgen de la Arrixaca
El Palmar, Murcia, 30120
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950
Hospital Universitario Ramon y Cajal
Madrid, 28034
Turkey (Türkiye)4 sites
Gazi University Medical Faculty Clinical Research Unit
Ankara, 06500
Akdeniz University Medical Faculty
Antalya, 07059
Izmir Dr. Behcet Uz Cocuk Hastaliklari ve Cerrahisi Egitim ve Arastirma Hastanesi
Izmir, 35210
Ondokuz Mayis Univ. Med. Fac.
Samsun, 55139
United States3 sites
UNC Hospitals
Chapel Hill, North Carolina, 27514
UC San Diego Health
La Jolla, California, 92024
Meridian Clinical Research
Norfolk, Virginia, 23502
Hungary3 sites
Semmelweis Egyetem
Budapest, 1083
Debreceni Egyetem
Debrecen, Hajdú-Bihar, 4032
Pecsi Tudomanyegyetem KK
Pécs, Baranya, 7623
Italy3 sites
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
Florence, 50139
Azienda Ospedaliera Universitaria 'Federico II'
Naples, 80131
Azienda Ospedaliera Universitaria- Università degli Studi della Campania "Luigi Vanvitelli"
Naples, 80138
Serbia3 sites
Clinic of Neurology and Psychiatry for Children and Youth
Belgrade, Balkans, 11000
University Children Hospital
Belgrade, Balkans, 11000
Mother and Child Health Care Institute of Serbia ,,Dr Vukan Cupic''
Belgrade, Balkans, 11070
Tunisia3 sites
Hopital Razi
Manouba, Mannouba, 2010
Hôpital Fattouma Bourghiba
Monastir, 5000
Hôpital Habib Bourguiba
Sfax, 3029
Belgium2 sites
Universitair Ziekenhuis Ghent
Ghent, East Flanders, 9000
Clinique CHC MontLégia
Liège, Wallonia, 4000
Greece2 sites
General Hospital of Larissa
Larissa, Thessaly, 41110
General Hospital of Thessaloniki 'Hippokration'
Thessaloniki, 54642
Israel2 sites
Hadassah University Hospital - Ein Kerem
Jerusalem, Levant, 9112001
Schneider Children's Medical Center
Petach-Tikva, Tel Aviv, 4920235
Saudi Arabia2 sites
King Faisal Specialist Hospital & Research Center
Jeddah, Mecca Region, 40047
King Saud University
Riyadh, 11461
Argentina1 site
Hospital Italiano de Buenos Aires
Ciudad Autonoma Buenos Aires, Buenos Aires, 1199
About this study
Official title: An Open-Label, Randomized, Multicenter, Active-Controlled, Parallel-Group Study to Evaluate the Safety, Tolerability, and Efficacy of BIIB017 in Pediatric Subjects Aged 10 to Less Than 18 Years for the Treatment of Relapsing-Remitting Multiple Sclerosis, With Optional Open-Label Extension
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
- Being tested
- BIIB017 (peginterferon beta-1a), Interferon beta type 1a
- Medical subject headings
- Multiple Sclerosis, Relapsing-Remitting
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BIIB017 (peginterferon beta-1a)Drug
Also called PLEGRIDY
Administered as specified in the treatment arm
Interferon beta type 1aDrug
Also called Avonex
Administered as specified in the treatment arm
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Interferon beta type 1a |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: From Week 96 to Week 196
Secondary outcomes (40)
Part 1: ARR at Week 96
An 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.
Measured: 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
Measured: 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
Measured: 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
Measured: Weeks 24, 48, and 96
Part 1: Number of Gd-Enhancing Lesions on Brain MRI Scans at Weeks 24, 48, and 96
Measured: Weeks 24, 48, and 96
Part 1: Time to First Relapse
Measured: Up to Week 96
Part 1: Percentage of Participants Free of Relapse at Weeks 48 and 96
Measured: Weeks 48 and 96
Part 1: Change from Baseline in Cognition at Weeks 24, 48, 72, and 96 as Measured by the Symbol Digit Modality Test (SDMT)
The SDMT measures the time to pair abstract symbols with specific numbers. The test requires elements of attention, visuoperceptual processing, working memory, and psychomotor speed. The score is the number of correctly coded items from 0 (worst) to 110 (best) in 90 seconds. The total score provides a measure of the speed and accuracy of symbol-digit substitution. Higher scores indicate better performance.
Measured: Baseline, Weeks 24, 48, 72, and 96
Part 1: Change from Baseline in the Expanded Disability Status Scale (EDSS) Score at Weeks 48 and 96
EDSS is based on a standardized neurological exam and focuses on symptoms that commonly occur in MS. Scores range from 0.0 (normal) to 10.0 (death due to MS).
Measured: Baseline, Weeks 48, and 96
Part 1: Change from Baseline in the Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Weeks 24, 48, 72 and 96
The PedsQL consists of 23 items in four generic score scales: physical functioning, emotional functioning, social functioning, and school functioning that measures health related quality of life. The questionnaire asks how much of a problem each item has been during the past month. Each item is answered on a scale of 0 (never) to 4 (almost always) then the scores are transformed to a 0 to 100 scale, so that higher scores indicate better heath related quality of life.
Measured: Baseline, Weeks 24, 48, 72 and 96
Part 1: Area Under the Plasma Concentration-Time Curve from Time Zero to End of Dosing Interval (AUCtau) for BIIB017
Measured: Within 8 hours postdose on Day 1 of Week 1; Within 8 hours, 48 and 120 hours postdose on Day 1 of Week 4; Within 8 hours postdose on Day 1 of Week 24
Part 1: Maximum Observed Plasma Concentration (Cmax) at Steady State for BIIB017
Measured: Within 8 hours postdose on Day 1 of Week 1; Within 8 hours, 48 and 120 hours postdose on Day 1 of Week 4; Within 8 hours postdose on Day 1 of Week 24
Part 1: Time to Reach Maximum Observed Plasma Concentration (Tmax) at Steady State for BIIB017
Measured: Within 8 hours postdose on Day 1 of Week 1; Within 8 hours, 48 and 120 hours postdose on Day 1 of Week 4; Within 8 hours postdose on Day 1 of Week 24
Part 1: 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.
Measured: Up to Week 100
Part 1: Change from Baseline in Height at Weeks 24, 48, 72, 96, and 100
Measured: Baseline, Weeks 24, 48, 72, 96, and 100
Part 1: Change from Baseline in Weight at Weeks 24, 48, 72, 96, and 100
Measured: Baseline, Weeks 24, 48, 72, 96, and 100
Part 1: Change from Baseline in Tanner Score at Weeks 24, 48, 72, 96, and 100
Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected for all male participants with bone age less than (<) 16 years and for female participants who are pre-menarche and have a bone age < 16 years and will be stopped once the participant's bone age reaches greater than or equal to (>=) 16 years or (once the participant is post-menarche. Tanner Score can be omitted if required by country-specific regulations and/or local ethics committees.
Measured: Baseline, Weeks 24, 48, 72, 96, and 100
Part 1: Number of Participants With Binding and Neutralizing Antibodies to Interferon Beta Type 1a (IFN β-1a) [All Participants]
Presence of IFN β-1a antibodies in human serum will be determined using enzyme-linked immunosorbent assay (ELISA) followed by further characterization and titration of positive samples in a cell-based neutralizing antibody assay.
Measured: Up to Week 96
Part 1: Number of Participants With Binding Antibodies to Peginterferon (PEG) [BIIB017-Treated Participants]
Anti-PEG binding antibodies in human serum will be determined using an ELISA.
Measured: Up to Week 96
Part 1: Change from Baseline in Depression as Assessed by Mini International Neuropsychiatric Interview for Children and Adolescents (MINI-KID) at Weeks 12, 24, 36, 48, 60, 72, 84, 96, and 100
MINI-KID is a short (approximately 15 minute) structured diagnostic interview used to assess the presence of 20 diagnostic and statistical manual of mental disorders, 4th Edition (DSM-IV) child and adolescent psychiatric disorders as well as the risk of suicide. The MINI-Kid frames questions in language that is easy for children and adolescents. It consists of 137 questions across 24 modules.
Measured: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Blood Pressure at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Measured: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Pulse Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Measured: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Body Temperature at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Measured: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Respiratory Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Measured: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Weeks 48, 96, and 100
Measured: Baseline (Before dosing), Weeks 48, 96, and 100
Part 1: Percentage of Participants with Changes Over Time in Clinical Laboratory Values
Clinical laboratory tests include: hematology, chemistry (including liver, renal and thyroid function), and coagulation tests.
Measured: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 2: ARR at Weeks 144 and 192
An 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.
Measured: Weeks 144 and 192
Part 2: Change from Baseline in EDSS Score at Weeks 120, 144, 168, 192, and 196
EDSS is based on a standardized neurological exam and focuses on symptoms that commonly occur in MS. Scores range from 0.0 (normal) to 10.0 (death due to MS).
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Height at Weeks 120, 144, 168, 192, and 196
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Weight at Weeks 120, 144, 168, 192, and 196
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Tanner Score at Weeks 120, 144, 168, 192, and 196
Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected for all male participants with bone age < 16 years and for female participants who are pre-menarche and have a bone age <16 years and will be stopped once the participant's bone age reaches >= 16 years or (once the participant is post-menarche. Tanner Score can be omitted if required by country-specific regulations and/or local ethics committees.
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Number of Participants With Binding and Neutralizing Antibodies to IFN β-1a (All Participants)
Presence of IFN β-1a antibodies in human serum will be determined using ELISA followed by further characterization and titration of positive samples in a cell-based neutralizing antibody assay.
Measured: Up to Week 192
Part 2: Number of Participants With Binding Antibodies to PEG (BIIB017-Treated Participants)
Anti-PEG binding antibodies in human serum will be determined using an ELISA.
Measured: Up to Week 192
Part 2: Change from Baseline in Blood Pressure at Weeks 120,144,168, 192, and 196
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Pulse Rate at Weeks 120, 144, 168, 192, and 196
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Body Temperature at Weeks 120, 144, 168, 192, and 196
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Respiratory Rate at Weeks 120, 144, 168, 192, and 196
Measured: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in 12-Lead ECG Parameters at Weeks 144, 192, and 196
Measured: Baseline (Week 96), Weeks 144, 192, and 196
Part 2: Change from Baseline in Depression as Assessed by Mini International Neuropsychiatric Interview for Children and Adolescents (MINI-KID) at Weeks 108, 120, 132, 144,156, 168, 180, 192, and 196
MINI-KID is a structured diagnostic interview instrument for psychiatric evaluation and outcome tracking. All questions are answered Yes or No.
Measured: Baseline (Week 96), Weeks 108, 120, 132, 144,156, 168, 180, 192, and 196
Part 2: Percentage of Participants with Changes Over Time in Clinical Laboratory Values
Clinical laboratory tests include: hematology, chemistry (including liver, renal and thyroid function), and coagulation tests.
Measured: Baseline (Week 96), Weeks 108, 120, 132, 144, 156, 168, 180, 192, and 196
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2019
- Study start18 Oct 2019
- Primary completion20 May 2027 (estimated)
- Study completion20 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple Sclerosis, Relapsing-RemittingStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 152 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03958877
- Sponsor's protocol ID
- 105MS306
- Other IDs
- 2018-003008-38, 2023-505624-56, 2023, 2018
- Status last verified
- Sep 2026
- First submitted
- 20 May 2019
- Last updated
- 24 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Randomized, Multicenter, Active-Controlled, Parallel-Group Study to Evaluate the Safety, Tolerability, and Efficacy of BIIB017 in Pediatric Subjects Aged 10 to Less Than 18 Years for the Treatment of Relapsing-Remitting Multiple Sclerosis, With Optional Open-Label Extension
- ClinicalTrials.gov ID
- NCT03958877
- Acronym / sponsor ID
- 105MS306
- Conditions
- Multiple Sclerosis, Relapsing-Remitting
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 152 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 18 Oct 2019 (actual)
- Primary completion
- 20 May 2027 (estimated)
- Study completion
- 20 May 2027 (estimated)
- First posted
- 22 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BIIB017 (peginterferon beta-1a) | Experimental | Drug: BIIB017 (peginterferon beta-1a)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. |
| Avonex | Active comparator | Drug: Interferon beta type 1aParticipants 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. |
Who may qualify
- Ages
- 10 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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).
- Participants who completed the study treatment in Part 1 (Week 96 Visit), as per protocol.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BIIB017 (peginterferon beta-1a)Also: PLEGRIDYAdministered as specified in the treatment arm | Drug | BIIB017 (peginterferon beta-1a) |
| Interferon beta type 1aAlso: AvonexAdministered as specified in the treatment arm | Drug | Avonex |
Primary outcome
Part 1: Annualized Relapse Rate (ARR) at Week 48
Time frame: 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
Time frame: 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.
40 secondary outcomes
Part 1: ARR at Week 96
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Weeks 24, 48, and 96
Part 1: Number of Gd-Enhancing Lesions on Brain MRI Scans at Weeks 24, 48, and 96
Time frame: Weeks 24, 48, and 96
Part 1: Time to First Relapse
Time frame: Up to Week 96
Part 1: Percentage of Participants Free of Relapse at Weeks 48 and 96
Time frame: Weeks 48 and 96
Part 1: Change from Baseline in Cognition at Weeks 24, 48, 72, and 96 as Measured by the Symbol Digit Modality Test (SDMT)
Time frame: Baseline, Weeks 24, 48, 72, and 96
Part 1: Change from Baseline in the Expanded Disability Status Scale (EDSS) Score at Weeks 48 and 96
Time frame: Baseline, Weeks 48, and 96
Part 1: Change from Baseline in the Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Weeks 24, 48, 72 and 96
Time frame: Baseline, Weeks 24, 48, 72 and 96
Part 1: Area Under the Plasma Concentration-Time Curve from Time Zero to End of Dosing Interval (AUCtau) for BIIB017
Time frame: Within 8 hours postdose on Day 1 of Week 1; Within 8 hours, 48 and 120 hours postdose on Day 1 of Week 4; Within 8 hours postdose on Day 1 of Week 24
Part 1: Maximum Observed Plasma Concentration (Cmax) at Steady State for BIIB017
Time frame: Within 8 hours postdose on Day 1 of Week 1; Within 8 hours, 48 and 120 hours postdose on Day 1 of Week 4; Within 8 hours postdose on Day 1 of Week 24
Part 1: Time to Reach Maximum Observed Plasma Concentration (Tmax) at Steady State for BIIB017
Time frame: Within 8 hours postdose on Day 1 of Week 1; Within 8 hours, 48 and 120 hours postdose on Day 1 of Week 4; Within 8 hours postdose on Day 1 of Week 24
Part 1: Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Study Treatment Discontinuation
Time frame: Up to Week 100
Part 1: Change from Baseline in Height at Weeks 24, 48, 72, 96, and 100
Time frame: Baseline, Weeks 24, 48, 72, 96, and 100
Part 1: Change from Baseline in Weight at Weeks 24, 48, 72, 96, and 100
Time frame: Baseline, Weeks 24, 48, 72, 96, and 100
Part 1: Change from Baseline in Tanner Score at Weeks 24, 48, 72, 96, and 100
Time frame: Baseline, Weeks 24, 48, 72, 96, and 100
Part 1: Number of Participants With Binding and Neutralizing Antibodies to Interferon Beta Type 1a (IFN β-1a) [All Participants]
Time frame: Up to Week 96
Part 1: Number of Participants With Binding Antibodies to Peginterferon (PEG) [BIIB017-Treated Participants]
Time frame: Up to Week 96
Part 1: Change from Baseline in Depression as Assessed by Mini International Neuropsychiatric Interview for Children and Adolescents (MINI-KID) at Weeks 12, 24, 36, 48, 60, 72, 84, 96, and 100
Time frame: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Blood Pressure at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Time frame: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Pulse Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Time frame: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Body Temperature at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Time frame: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in Respiratory Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Time frame: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 1: Change from Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Weeks 48, 96, and 100
Time frame: Baseline (Before dosing), Weeks 48, 96, and 100
Part 1: Percentage of Participants with Changes Over Time in Clinical Laboratory Values
Time frame: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Part 2: ARR at Weeks 144 and 192
Time frame: Weeks 144 and 192
Part 2: Change from Baseline in EDSS Score at Weeks 120, 144, 168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Height at Weeks 120, 144, 168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Weight at Weeks 120, 144, 168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Tanner Score at Weeks 120, 144, 168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Number of Participants With Binding and Neutralizing Antibodies to IFN β-1a (All Participants)
Time frame: Up to Week 192
Part 2: Number of Participants With Binding Antibodies to PEG (BIIB017-Treated Participants)
Time frame: Up to Week 192
Part 2: Change from Baseline in Blood Pressure at Weeks 120,144,168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Pulse Rate at Weeks 120, 144, 168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Body Temperature at Weeks 120, 144, 168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in Respiratory Rate at Weeks 120, 144, 168, 192, and 196
Time frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Part 2: Change from Baseline in 12-Lead ECG Parameters at Weeks 144, 192, and 196
Time frame: Baseline (Week 96), Weeks 144, 192, and 196
Part 2: Change from Baseline in Depression as Assessed by Mini International Neuropsychiatric Interview for Children and Adolescents (MINI-KID) at Weeks 108, 120, 132, 144,156, 168, 180, 192, and 196
Time frame: Baseline (Week 96), Weeks 108, 120, 132, 144,156, 168, 180, 192, and 196
Part 2: Percentage of Participants with Changes Over Time in Clinical Laboratory Values
Time frame: Baseline (Week 96), Weeks 108, 120, 132, 144, 156, 168, 180, 192, and 196
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2026.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
65 sites in 22 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BIIB017 (peginterferon beta-1a) and Interferon beta type 1a in people with Multiple Sclerosis, Relapsing-Remitting, run by Biogen at 65 sites in 22 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 24 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 65 sites in Russia, France, Portugal, Croatia, Germany and 17 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part 1: Annualized Relapse Rate (ARR) at Week 48 (time frame: Week 48). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.