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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03958877




Registration number
NCT03958877
Ethics application status
Date submitted
20/05/2019
Date registered
22/05/2019

Titles & IDs
Public title
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
Scientific 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
Secondary ID [1] 0 0
2018-003008-38
Secondary ID [2] 0 0
105MS306
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Sclerosis, Relapsing-Remitting 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BIIB017 (peginterferon beta-1a)
Treatment: Drugs - Interferon beta type 1a

Experimental: 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.

Active comparator: Avonex - 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.


Treatment: Drugs: BIIB017 (peginterferon beta-1a)
Administered as specified in the treatment arm

Treatment: Drugs: Interferon beta type 1a
Administered as specified in the treatment arm

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part 1: Annualized Relapse Rate (ARR) at Week 48
Timepoint [1] 0 0
Week 48
Primary outcome [2] 0 0
Part 2: Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Treatment Discontinuation
Timepoint [2] 0 0
From Week 96 to Week 196
Secondary outcome [1] 0 0
Part 1: ARR at Week 96
Timepoint [1] 0 0
Week 96
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Weeks 24, 48, and 96
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
Weeks 24, 48, and 96
Secondary outcome [4] 0 0
Part 1: Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain MRI Scans at Weeks 24, 48, and 96
Timepoint [4] 0 0
Weeks 24, 48, and 96
Secondary outcome [5] 0 0
Part 1: Number of Gd-Enhancing Lesions on Brain MRI Scans at Weeks 24, 48, and 96
Timepoint [5] 0 0
Weeks 24, 48, and 96
Secondary outcome [6] 0 0
Part 1: Time to First Relapse
Timepoint [6] 0 0
Up to Week 96
Secondary outcome [7] 0 0
Part 1: Percentage of Participants Free of Relapse at Weeks 48 and 96
Timepoint [7] 0 0
Weeks 48 and 96
Secondary outcome [8] 0 0
Part 1: Change from Baseline in Cognition at Weeks 24, 48, 72, and 96 as Measured by the Symbol Digit Modality Test (SDMT)
Timepoint [8] 0 0
Baseline, Weeks 24, 48, 72, and 96
Secondary outcome [9] 0 0
Part 1: Change from Baseline in the Expanded Disability Status Scale (EDSS) Score at Weeks 48 and 96
Timepoint [9] 0 0
Baseline, Weeks 48, and 96
Secondary outcome [10] 0 0
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
Timepoint [10] 0 0
Baseline, Weeks 24, 48, 72 and 96
Secondary outcome [11] 0 0
Part 1: Area Under the Plasma Concentration-Time Curve from Time Zero to End of Dosing Interval (AUCtau) for BIIB017
Timepoint [11] 0 0
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
Secondary outcome [12] 0 0
Part 1: Maximum Observed Plasma Concentration (Cmax) at Steady State for BIIB017
Timepoint [12] 0 0
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
Secondary outcome [13] 0 0
Part 1: Time to Reach Maximum Observed Plasma Concentration (Tmax) at Steady State for BIIB017
Timepoint [13] 0 0
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
Secondary outcome [14] 0 0
Part 1: Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Study Treatment Discontinuation
Timepoint [14] 0 0
Up to Week 100
Secondary outcome [15] 0 0
Part 1: Change from Baseline in Height at Weeks 24, 48, 72, 96, and 100
Timepoint [15] 0 0
Baseline, Weeks 24, 48, 72, 96, and 100
Secondary outcome [16] 0 0
Part 1: Change from Baseline in Weight at Weeks 24, 48, 72, 96, and 100
Timepoint [16] 0 0
Baseline, Weeks 24, 48, 72, 96, and 100
Secondary outcome [17] 0 0
Part 1: Change from Baseline in Tanner Score at Weeks 24, 48, 72, 96, and 100
Timepoint [17] 0 0
Baseline, Weeks 24, 48, 72, 96, and 100
Secondary outcome [18] 0 0
Part 1: Number of Participants With Binding and Neutralizing Antibodies to Interferon Beta Type 1a (IFN ß-1a) [All Participants]
Timepoint [18] 0 0
Up to Week 96
Secondary outcome [19] 0 0
Part 1: Number of Participants With Binding Antibodies to Peginterferon (PEG) [BIIB017-Treated Participants]
Timepoint [19] 0 0
Up to Week 96
Secondary outcome [20] 0 0
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
Timepoint [20] 0 0
Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, and 100
Secondary outcome [21] 0 0
Part 1: Change from Baseline in Blood Pressure at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Timepoint [21] 0 0
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Secondary outcome [22] 0 0
Part 1: Change from Baseline in Pulse Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Timepoint [22] 0 0
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Secondary outcome [23] 0 0
Part 1: Change from Baseline in Body Temperature at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Timepoint [23] 0 0
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Secondary outcome [24] 0 0
Part 1: Change from Baseline in Respiratory Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Timepoint [24] 0 0
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Secondary outcome [25] 0 0
Part 1: Change from Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Weeks 48, 96, and 100
Timepoint [25] 0 0
Baseline (Before dosing), Weeks 48, 96, and 100
Secondary outcome [26] 0 0
Part 1: Percentage of Participants with Changes Over Time in Clinical Laboratory Values
Timepoint [26] 0 0
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100
Secondary outcome [27] 0 0
Part 2: ARR at Weeks 144 and 192
Timepoint [27] 0 0
Weeks 144 and 192
Secondary outcome [28] 0 0
Part 2: Change from Baseline in EDSS Score at Weeks 120, 144, 168, 192, and 196
Timepoint [28] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [29] 0 0
Part 2: Change from Baseline in Height at Weeks 120, 144, 168, 192, and 196
Timepoint [29] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [30] 0 0
Part 2: Change from Baseline in Weight at Weeks 120, 144, 168, 192, and 196
Timepoint [30] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [31] 0 0
Part 2: Change from Baseline in Tanner Score at Weeks 120, 144, 168, 192, and 196
Timepoint [31] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [32] 0 0
Part 2: Number of Participants With Binding and Neutralizing Antibodies to IFN ß-1a (All Participants)
Timepoint [32] 0 0
Up to Week 192
Secondary outcome [33] 0 0
Part 2: Number of Participants With Binding Antibodies to PEG (BIIB017-Treated Participants)
Timepoint [33] 0 0
Up to Week 192
Secondary outcome [34] 0 0
Part 2: Change from Baseline in Blood Pressure at Weeks 120,144,168, 192, and 196
Timepoint [34] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [35] 0 0
Part 2: Change from Baseline in Pulse Rate at Weeks 120, 144, 168, 192, and 196
Timepoint [35] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [36] 0 0
Part 2: Change from Baseline in Body Temperature at Weeks 120, 144, 168, 192, and 196
Timepoint [36] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [37] 0 0
Part 2: Change from Baseline in Respiratory Rate at Weeks 120, 144, 168, 192, and 196
Timepoint [37] 0 0
Baseline (Week 96), Weeks 120, 144, 168, 192, and 196
Secondary outcome [38] 0 0
Part 2: Change from Baseline in 12-Lead ECG Parameters at Weeks 144, 192, and 196
Timepoint [38] 0 0
Baseline (Week 96), Weeks 144, 192, and 196
Secondary outcome [39] 0 0
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
Timepoint [39] 0 0
Baseline (Week 96), Weeks 108, 120, 132, 144,156, 168, 180, 192, and 196
Secondary outcome [40] 0 0
Part 2: Percentage of Participants with Changes Over Time in Clinical Laboratory Values
Timepoint [40] 0 0
Baseline (Week 96), Weeks 108, 120, 132, 144, 156, 168, 180, 192, and 196

Eligibility
Key inclusion criteria
Key

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
Minimum age
10 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Part 1:

* Primary progressive, secondary progressive, or progressive relapsing. 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 ß-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"

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
North Carolina
Country [3] 0 0
United States of America
State/province [3] 0 0
Virginia
Country [4] 0 0
Argentina
State/province [4] 0 0
Buenos Aires
Country [5] 0 0
Belgium
State/province [5] 0 0
East Flanders
Country [6] 0 0
Belgium
State/province [6] 0 0
Wallonia
Country [7] 0 0
Bulgaria
State/province [7] 0 0
Sofia
Country [8] 0 0
Croatia
State/province [8] 0 0
Dalmatia
Country [9] 0 0
Croatia
State/province [9] 0 0
Zagreb
Country [10] 0 0
Czechia
State/province [10] 0 0
Hradec Kralove
Country [11] 0 0
France
State/province [11] 0 0
Bas Rhin
Country [12] 0 0
France
State/province [12] 0 0
Haute Garonne
Country [13] 0 0
France
State/province [13] 0 0
Herault
Country [14] 0 0
France
State/province [14] 0 0
Nord
Country [15] 0 0
France
State/province [15] 0 0
Val De Marne
Country [16] 0 0
Germany
State/province [16] 0 0
Baden Wuerttemberg
Country [17] 0 0
Germany
State/province [17] 0 0
Niedersachsen
Country [18] 0 0
Germany
State/province [18] 0 0
Nordrhein Westfalen
Country [19] 0 0
Germany
State/province [19] 0 0
Berlin
Country [20] 0 0
Greece
State/province [20] 0 0
Thessaly
Country [21] 0 0
Greece
State/province [21] 0 0
Thessaloniki
Country [22] 0 0
Hungary
State/province [22] 0 0
Baranya
Country [23] 0 0
Hungary
State/province [23] 0 0
Hajdú-Bihar
Country [24] 0 0
Hungary
State/province [24] 0 0
Budapest
Country [25] 0 0
Israel
State/province [25] 0 0
Levant
Country [26] 0 0
Israel
State/province [26] 0 0
Tel Aviv
Country [27] 0 0
Italy
State/province [27] 0 0
Firenze
Country [28] 0 0
Italy
State/province [28] 0 0
Napoli
Country [29] 0 0
Kuwait
State/province [29] 0 0
Shuwaikh
Country [30] 0 0
Portugal
State/province [30] 0 0
Lisbon
Country [31] 0 0
Portugal
State/province [31] 0 0
Minho
Country [32] 0 0
Portugal
State/province [32] 0 0
Coimbra
Country [33] 0 0
Portugal
State/province [33] 0 0
Porto
Country [34] 0 0
Russian Federation
State/province [34] 0 0
Bashkortostan
Country [35] 0 0
Russian Federation
State/province [35] 0 0
Krasnoyarsk Krai
Country [36] 0 0
Russian Federation
State/province [36] 0 0
Leningrad
Country [37] 0 0
Russian Federation
State/province [37] 0 0
Oblast
Country [38] 0 0
Russian Federation
State/province [38] 0 0
Siberia
Country [39] 0 0
Russian Federation
State/province [39] 0 0
Belgorod
Country [40] 0 0
Russian Federation
State/province [40] 0 0
Moscow
Country [41] 0 0
Russian Federation
State/province [41] 0 0
Rostov-on-Don
Country [42] 0 0
Russian Federation
State/province [42] 0 0
Yaroslavl
Country [43] 0 0
Saudi Arabia
State/province [43] 0 0
Makkah
Country [44] 0 0
Saudi Arabia
State/province [44] 0 0
Riyadh
Country [45] 0 0
Serbia
State/province [45] 0 0
Balkans
Country [46] 0 0
Slovakia
State/province [46] 0 0
Bratislava
Country [47] 0 0
Spain
State/province [47] 0 0
Barcelona
Country [48] 0 0
Spain
State/province [48] 0 0
Murcia
Country [49] 0 0
Spain
State/province [49] 0 0
Cordoba
Country [50] 0 0
Spain
State/province [50] 0 0
Madrid
Country [51] 0 0
Tunisia
State/province [51] 0 0
Mannouba
Country [52] 0 0
Tunisia
State/province [52] 0 0
Monastir
Country [53] 0 0
Tunisia
State/province [53] 0 0
Sfax
Country [54] 0 0
Turkey
State/province [54] 0 0
Ankara
Country [55] 0 0
Turkey
State/province [55] 0 0
Antalya
Country [56] 0 0
Turkey
State/province [56] 0 0
Izmir
Country [57] 0 0
Turkey
State/province [57] 0 0
Samsun

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biogen
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
US Biogen Clinical Trial Center
Address 0 0
Country 0 0
Phone 0 0
866-633-4636
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.