peginterferon beta-1a for Relapsing-remitting multiple sclerosis

Inclusion criteria

• {"criterion_text":"- [Part I] Must have a diagnosis of RRMS as defined by the revised consensus definition for pediatric MS\n- [Part I] Must have an EDSS score between 0.0 and 5.5\n- [Part I] 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).\n- [Part II] Participants who completed the study treatment in Part 1 (Week 96 Visit), as per protocol."}

Exclusion criteria

• {"criterion_text":"- [Part I] 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 subjects by the lack of clinically stable periods or clinical improvement.\n- [Part I] History of severe allergic or anaphylactic reactions or known drug hypersensitivity.\n- Known allergy to any component of Avonex or BIIB017 formulation.\n- 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).\n- [Part I] Any previous treatment with PEGylated human IFN β-1a\n- [Part II] Any significant changes in medical history occurring after enrolment 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.\n- [Part II] The subject could not tolerate BIIB017 in Part 1. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply."}

Primary endpoints

• {"endpoint_text":"- Part 1: the annualized relapse rate (ARR) at Week 48.","definition_or_measurement_approach":"Annualized relapse rate (ARR) measured at Week 48."}
• {"endpoint_text":"- Part 2: Percentage of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs leading to Study Treatment Discontinuation. [From Week 96 to week 196]","definition_or_measurement_approach":"Percentage of participants experiencing AEs, SAEs, and AEs leading to treatment discontinuation during Week 96 to Week 196."}

Secondary endpoints

• {"endpoint_text":"- [Part I] ARR at week 48","definition_or_measurement_approach":""}
• {"endpoint_text":"- [Part I] 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","definition_or_measurement_approach":"Proportion free of new or newly enlarging T2 hyperintense lesions on brain MRI at Weeks 24, 48, 96"}
• {"endpoint_text":"- [Part I] 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","definition_or_measurement_approach":"Proportion free of Gd-enhancing lesions and new/newly enlarging T2 lesions on brain MRI at Weeks 24, 48, 96"}
• {"endpoint_text":"- [Part I] Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain MRI Scans at Weeks 24, 48, and 96","definition_or_measurement_approach":"Count of new or newly enlarging T2 hyperintense lesions on brain MRI at Weeks 24, 48, 96"}
• {"endpoint_text":"- [Part I] Number of Gd-Enhancing Lesions on Brain MRI Scans at Weeks 24, 48, and 96","definition_or_measurement_approach":"Count of gadolinium-enhancing lesions on brain MRI at Weeks 24, 48, 96"}
• {"endpoint_text":"- [Part I] Time to First Relapse (timeframe: Up to week 96).","definition_or_measurement_approach":"Time from randomization to first documented relapse up to Week 96"}
• {"endpoint_text":"- [Part I] Percentage of Participants Free of Relapse at Weeks 48 and 96.","definition_or_measurement_approach":"Proportion of participants without relapse at Weeks 48 and 96"}
• {"endpoint_text":"- [Part I] Change from Baseline in Cognition at Weeks 24, 48, 72, and 96 as Measured by the Symbol Digit Modality Test (SDMT)","definition_or_measurement_approach":"Change from baseline in SDMT score at Weeks 24, 48, 72, 96"}
• {"endpoint_text":"- [Part I] Change from Baseline in the Expanded Disability Status Scale (EDSS) Score at Weeks 48 and 96","definition_or_measurement_approach":"Change from baseline in EDSS score at Weeks 48 and 96"}
• {"endpoint_text":"- [Part I] 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","definition_or_measurement_approach":"Change from baseline in PedsQL at Weeks 24, 48, 72, 96"}
• {"endpoint_text":"- [Part I] 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]","definition_or_measurement_approach":"AUCtau for BIIB017 measured at specified postdose timepoints (Week 1 Day1 within 8h; Week4 Day1 within 8, 48, 120h; Week24 Day1 within 8h)"}
• {"endpoint_text":"- [Part I] Part 1: Maximum Observed Plasma Concentration (Cmax) at Steady State for BIIB017 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","definition_or_measurement_approach":"Cmax for BIIB017 measured at specified postdose timepoints"}
• {"endpoint_text":"- [Part I] Time to Reach Maximum Observed Plasma Concentration (Tmax) at Steady State for BIIB017 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","definition_or_measurement_approach":"Tmax for BIIB017 measured at specified postdose timepoints"}
• {"endpoint_text":"- [Part I] Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Study Treatment Discontinuation (timeframe up to week 100)","definition_or_measurement_approach":"Percentage of participants with AEs, SAEs, and treatment-discontinuing AEs up to Week 100"}
• {"endpoint_text":"- [Part I] Change from Baseline in Height at Weeks 24, 48, 72, 96, and 100","definition_or_measurement_approach":"Change from baseline in height at Weeks 24, 48, 72, 96, 100"}
• {"endpoint_text":"- [Part I] Change from Baseline in Weight at Weeks 24, 48, 72, 96, and 100","definition_or_measurement_approach":"Change from baseline in weight at Weeks 24, 48, 72, 96, 100"}
• {"endpoint_text":"- [Part I] Number of Participants With Binding and Neutralizing Antibodies to Interferon Beta Type 1a (IFN β-1a) [All Participants] [Time Frame: Up to Week 96]","definition_or_measurement_approach":"Count of participants with binding and neutralizing antibodies to IFN β-1a up to Week 96"}
• {"endpoint_text":"- [Part I] 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 (Timeframe: Up to Week 96)","definition_or_measurement_approach":"Count of BIIB017-treated participants with anti-PEG binding antibodies determined by ELISA up to Week 96"}
• {"endpoint_text":"- [Part I] 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","definition_or_measurement_approach":"Change from baseline in depression per MINI-KID at listed weeks"}
• {"endpoint_text":"- [Part I] Change from Baseline in Blood Pressure at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 10","definition_or_measurement_approach":"Change from baseline in blood pressure at listed weeks"}
• {"endpoint_text":"- [Part I] Change from Baseline in Pulse Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100","definition_or_measurement_approach":"Change from baseline in pulse rate at listed weeks"}
• {"endpoint_text":"- [Part I] Change from Baseline in Body Temperature at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100","definition_or_measurement_approach":"Change from baseline in body temperature at listed weeks"}
• {"endpoint_text":"- [Part I] Change from Baseline in Respiratory Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100","definition_or_measurement_approach":"Change from baseline in respiratory rate at listed weeks"}
• {"endpoint_text":"- [Part I] Change from Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Weeks 48, 96, and 100","definition_or_measurement_approach":"Change from baseline in 12-lead ECG parameters at Weeks 48, 96, 100"}
• {"endpoint_text":"- [Part I] Percentage of Participants with Changes Over Time in Clinical Laboratory Values at Baseline & Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 100","definition_or_measurement_approach":"Percentage of participants with lab value changes over time at listed visits"}
• {"endpoint_text":"- Part 2: ARR at Weeks 144 and 192","definition_or_measurement_approach":"Annualized relapse rate at Weeks 144 and 192"}
• {"endpoint_text":"- Part 2: Change from Baseline in EDSS Score at Weeks 120, 144, 168, 192, and 196","definition_or_measurement_approach":"Change from baseline in EDSS at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Change from Baseline in Height at Weeks 120, 144, 168, 192, and 196","definition_or_measurement_approach":"Change from baseline in height at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Change from Baseline in Weight at Weeks 120, 144, 168, 192, and 196","definition_or_measurement_approach":"Change from baseline in weight at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Change from Baseline in Tanner Score at Weeks 120, 144, 168, 192, and 196","definition_or_measurement_approach":"Change from baseline in Tanner score at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Number of Participants With Binding and Neutralizing Antibodies to IFN β-1a (All Participants) (Time Frame: Up to week 192)","definition_or_measurement_approach":"Count of participants with binding and neutralizing antibodies to IFN β-1a up to Week 192"}
• {"endpoint_text":"- Part 2: Number of Participants With Binding Antibodies to PEG (BIIB017-Treated Participants) (Time Frame: Up to week 192)","definition_or_measurement_approach":"Count of BIIB017-treated participants with anti-PEG binding antibodies up to Week 192"}
• {"endpoint_text":"- Part 2: Change from Baseline in Blood Pressure at Weeks 120,144,168, 192, and 196","definition_or_measurement_approach":"Change from baseline in blood pressure at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Change from Baseline in Pulse Rate at Weeks 120, 144, 168, 192, and 196","definition_or_measurement_approach":"Change from baseline in pulse rate at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Change from Baseline in Body Temperature at Weeks 120, 144, 168, 192, and 196","definition_or_measurement_approach":"Change from baseline in body temperature at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Change from Baseline in Respiratory Rate at Weeks 120, 144, 168, 192, and 196","definition_or_measurement_approach":"Change from baseline in respiratory rate at Weeks 120, 144, 168, 192, 196"}
• {"endpoint_text":"- Part 2: Change from Baseline in 12-Lead ECG Parameters at Weeks 144, 192, and 196","definition_or_measurement_approach":"Change from baseline in 12-lead ECG parameters at Weeks 144, 192, 196"}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Change from baseline in depression per MINI-KID at listed weeks"}
• {"endpoint_text":"- Part 2: Percentage of Participants with Changes Over Time in Clinical Laboratory Values at Baseline & (Week 96), Weeks 108, 120, 132, 144, 156, 168, 180, 192, and 196","definition_or_measurement_approach":"Percentage of participants with lab value changes over time at listed visits including Week 96 baseline comparator"}

Bulgaria

Earliest CTIS Part Ii Submission Date

30-09-2024

Latest Decision Or Authorization Date

08-11-2024

Processing Time Days

39

Number Of Sites

1

Number Of Participants

7

Croatia

Earliest CTIS Part Ii Submission Date

30-09-2024

Latest Decision Or Authorization Date

05-11-2024

Processing Time Days

36

Number Of Sites

1

Number Of Participants

12

Czechia

Earliest CTIS Part Ii Submission Date

30-09-2024

Latest Decision Or Authorization Date

08-11-2024

Processing Time Days

39

Number Of Sites

1

Number Of Participants

2

Slovakia

Earliest CTIS Part Ii Submission Date

30-09-2024

Latest Decision Or Authorization Date

05-11-2024

Processing Time Days

36

Number Of Sites

1

Number Of Participants

2

Hungary

Earliest CTIS Part Ii Submission Date

30-09-2024

Latest Decision Or Authorization Date

11-09-2025

Processing Time Days

346

Number Of Sites

1

Number Of Participants

6

Primary sponsor

Full Name

Biogen Idec Research Limited

Organisation Type

Pharmaceutical company

Country Of Registered Address

United Kingdom

Contract research organisations

Name

Fortrea Inc.

Responsibilities

15 - Home health services

Name

IQVIA Limited

Responsibilities

1,12,13,2,5,6

Name

PPD (UK) Limited

Responsibilities

8

Name

Icon Clinical Research Limited

Responsibilities

15 - Rating Scale Management: Licensing and Translations

Name

Medidata Solutions Inc.

Responsibilities

7

Name

Signant Health Global LLC

Responsibilities

3

Name

WCG Clinical Inc.

Responsibilities

15 - Rater training and certification

Third parties

• {"country":"United States","full_name":"Quest Diagnostics Nichols Institute Inc.","duties_or_roles":"4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"Fisher Clinical Services UK Limited","duties_or_roles":"14","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"15 - Home health services","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Neurorx Research Inc.","duties_or_roles":"15 - Medical image analysis/ review - X-ray, MRI, ultrasound, etc.","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"15 - Sample storage","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Neurostatus-UHB AG","duties_or_roles":"15 - Rating Scales Training","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"1,12,13,2,5,6","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"3","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"PPD (UK) Limited","duties_or_roles":"8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"15 - Rating Scale Management: Licensing and Translations","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"15 - Equipment supplier","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"15 - Patient reimbursement","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"4","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"15 - Equipment supplier","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"15 - Rater training and certification","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC (duplicate entry)","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}