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

• Register: ClinicalTrials.gov
• Last refreshed on: 22 January 2024
• Main ID: NCT03958877
• Date of registration: 20/05/2019
• Prospective Registration: Yes
• Primary sponsor: Biogen
• 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
• Date of first enrolment: October 18, 2019
• Target sample size: 142
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03958877
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Bulgaria, Croatia, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Kuwait, Portugal, Russian Federation, Saudi Arabia, Serbia, Slovakia, Spain, Tunisia, Turkey, United States

Contacts

• Name: US Biogen Clinical Trial Center

• Name: Medical Director
• Affiliation: Biogen

Key inclusion & exclusion criteria

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. 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"



Age minimum: 10 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple Sclerosis, Relapsing-Remitting

Intervention(s)

Drug: Interferon beta type 1a

Drug: BIIB017 (peginterferon beta-1a)

Primary Outcome(s)

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]

Part 1: Annualized Relapse Rate (ARR) at Week 48 [Time Frame: Week 48]

Secondary Outcome(s)

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: Number of Participants With Binding Antibodies to Peginterferon (PEG) [BIIB017-Treated Participants] [Time Frame: Up to Week 96]

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: 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 1: ARR at Week 96 [Time Frame: Week 96]

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 the Expanded Disability Status Scale (EDSS) Score at Weeks 48 and 96 [Time Frame: Baseline, Weeks 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: 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 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 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 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 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: Percentage of Participants Free of Relapse at Weeks 48 and 96 [Time Frame: Weeks 48 and 96]

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: 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 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 Tanner Score at Weeks 24, 48, 72, 96, and 100 [Time Frame: Baseline, Weeks 24, 48, 72, 96, and 100]

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 2: Number of Participants With Binding Antibodies to PEG (BIIB017-Treated Participants) [Time Frame: Up to Week 192]

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 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 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 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: Number of Participants With Binding and Neutralizing Antibodies to IFN ß-1a (All Participants) [Time Frame: Up to Week 192]

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]

Part 1: Time to First Relapse [Time Frame: Up to Week 96]

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: 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 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 Height at Weeks 24, 48, 72, 96, and 100 [Time Frame: Baseline, Weeks 24, 48, 72, 96, and 100]

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: 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 Participants With Binding and Neutralizing Antibodies to Interferon Beta Type 1a (IFN ß-1a) [All Participants] [Time Frame: Up to Week 96]

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 2: ARR at Weeks 144 and 192 [Time Frame: Weeks 144 and 192]

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 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 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 Weight at Weeks 120, 144, 168, 192, and 196 [Time Frame: Baseline (Week 96), Weeks 120, 144, 168, 192, and 196]

Secondary ID(s)

2023-505624-56

105MS306

2018-003008-38

Source(s) of Monetary Support

Please refer to primary and secondary sponsors