Study Summary
This trial will study a new MS drug in children to see if it is safe and effective. Part 1 will assess how the drug works in the body, and Part 2 will evaluate long-term safety and effectiveness.
Eligible Conditions
- Multiple Sclerosis, Relapsing-Remitting
Treatment Effectiveness
Phase-Based Effectiveness
Phase 3
Study Objectives
2 Primary · 40 Secondary · Reporting Duration: Baseline (Week 96), Weeks 108, 120, 132, 144,156, 168, 180, 192 and 196
Week 48
Part 1: Change from Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Weeks 48, 96 and 100
Week 108
Part 2: Percentage of Participants with Changes Over Time in Clinical Laboratory Values
Week 108
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
Week 120
Part 2: Change from Baseline in Blood Pressure at Weeks 120,144,168,192 and 196
Part 2: Change from Baseline in Body Temperature at Weeks 120, 144, 168, 192 and 196
Part 2: Change from Baseline in EDSS Score at Weeks 120, 144, 168, 192 and 196
Part 2: Change from Baseline in Heart Rate at Weeks 120, 144, 168, 192 and 196
Part 2: Change from Baseline in Height at Weeks 120, 144, 168, 192 and 196
Part 2: Change from Baseline in Respiratory Rate at Weeks 120, 144, 168, 192 and 196
Part 2: Change from Baseline in Tanner Score at Weeks 120, 144, 168, 192 and 196
Part 2: Change from Baseline in Weight at Weeks 120, 144, 168, 192 and 196
Week 144
Part 2: Change from Baseline in 12-Lead ECG Parameters at Weeks 144, 192 and 196
Week 12
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
Week 24
Part 1: Change from Baseline in Cognition at Weeks 24, 48, 72 and 96 as Measured by the Symbol Digit Modality Test (SDMT)
Week 24
Part 1: Change from Baseline in Height at Weeks 24, 48, 72, 96 and 100
Part 1: Change from Baseline in Tanner Score at Weeks 24, 48, 72, 96 and 100
Part 1: Change from Baseline in Weight at Weeks 24, 48, 72, 96 and 100
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, 96 and 100
Week 4
Part 1: Change from Baseline in Blood Pressure at 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
Part 1: Change from Baseline in Heart Rate at 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
Week 4
Part 1: Change from Baseline in Respiratory Rate at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96 and 100
Week 48
Part 1: Change from Baseline in the Expanded Disability Status Scale (EDSS) Score at Weeks 48 and 96
Week 196
Part 2: Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Study Treatment Discontinuation
Up to Week 100
Part 1: Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Study Treatment Discontinuation
Up to Week 192
Part 2: Number of Participants With Binding Antibodies to PEG (BIIB017-Treated Participants)
Part 2: Number of Participants With Binding and Neutralizing Antibodies to IFN β-1a (All Participants)
Up to Week 96
Part 1: Number of Participants With Binding Antibodies to Peginterferon (PEG) [BIIB017-Treated Participants]
Part 1: Number of Participants With Binding and Neutralizing Antibodies to Interferon Beta Type 1a (IFN β-1a) [All Participants]
Part 1: Percentage of Participants Free of Relapse Up to Week 96
Part 1: Time to First Relapse
Week 48
Part 1: ARR at Week 48
Week 96
Part 1: Annualized Relapse Rate (ARR) at Week 96
Weeks 144 and 192
Part 2: ARR at Weeks 144 and 192
Week 24
Part 1: Number of Gd-Enhancing Lesions on Brain MRI Scans at 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
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
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
Week 24
Part 1: Area Under the Plasma Concentration-Time Curve from Time Zero to End of Dosing Interval (AUCtau) for BIIB017
Part 1: Maximum Observed Plasma Concentration (Cmax) at Steady State for BIIB017
Part 1: Time to Reach Maximum Observed Plasma Concentration (Tmax) at Steady State for BIIB017
Trial Safety
Phase-Based Safety
This is further along than 85% of similar trials
Side Effects for
Year 1: Peginterferon Beta-1a Q4W
56%Injection Site Erythema
47%Influenza Like Illness
44%Pyrexia
41%Headache
22%Multiple Sclerosis Relapse
19%Myalgia
18%Chills
14%Nasopharyngitis
14%Asthenia
13%Back Pain
13%Injection Site Pain
11%Arthralgia
11%Pain In Extremity
11%Injection Site Pruritus
11%Fatigue
9%Nausea
7%Body Temperature Increased
7%Vomiting
6%Urinary Tract Infection
6%Pain
6%Vertigo
6%Hypoaesthesia
5%Cough
5%Hyperthermia
5%Depression
5%Oropharyngeal Pain
5%Muscular Weakness
4%Insomnia
4%Alanine Aminotransferase Increased
4%Dizziness
4%Paraesthesia
4%Diarrhoea
3%Upper Respiratory Tract Infection
Awards & Highlights
No Placebo Group
All patients enrolled in this trial will receive the new treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Trial Design
2 Treatment Groups
Avonex
1 of 2
BIIB017 (peginterferon beta-1a)
1 of 2
Active Control
Experimental Treatment
142 Total Participants · 2 Treatment Groups
Primary Treatment: BIIB017 (peginterferon beta-1a) · No Placebo Group · Phase 3
BIIB017 (peginterferon beta-1a)
Drug
Experimental Group · 1 Intervention: BIIB017 (peginterferon beta-1a) · Intervention Types: DrugAvonex
Drug
ActiveComparator Group · 1 Intervention: Interferon beta type 1a · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Peginterferon beta-1a
FDA approved
Trial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline (week 96), weeks 108, 120, 132, 144,156, 168, 180, 192 and 196
Who is running the clinical trial?
BiogenLead Sponsor
567 Previous Clinical Trials
456,002 Total Patients Enrolled
224 Trials studying Multiple Sclerosis
141,135 Patients Enrolled for Multiple Sclerosis
Medical DirectorStudy DirectorBiogen
2,670 Previous Clinical Trials
7,959,070 Total Patients Enrolled
134 Trials studying Multiple Sclerosis
124,947 Patients Enrolled for Multiple Sclerosis
Eligibility Criteria
Age 10 - 18 · All Participants · 4 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:You have been diagnosed with RRMS using the new guidelines for pediatric MS.
You must have a mild to moderate level of disability as measured by the EDSS score between 0.0 and 5.5.
You have already finished the first part of the study and completed the 96-week treatment according to the study's plan.
You have had at least one relapse in the past year or two relapses in the past two years, or have signs of disease activity on a recent brain MRI even if you have no symptoms.
Frequently Asked Questions
Are the requirements for this trial limiting it to a specific age group?
"This particular research study is focused on children aged 10-18." - Anonymous Online Contributor
Unverified Answer
Am I an eligible candidate for this research?
"The current clinical trial has a set of inclusion criteria that requires potential participants to have multiple sclerosis, be in an acute relapsing state, and be between the ages of 10 and 18. Up to 142 patients will be accepted into the trial." - Anonymous Online Contributor
Unverified Answer
Are there any risks associated with BIIB017 (peginterferon beta-1a)?
"BIIB017 (peginterferon beta-1a) has been studied in Phase 3 trials, meaning that there is both safety and efficacy data available. Our team rates it as a 3 on our safety scale." - Anonymous Online Contributor
Unverified Answer
What purpose does BIIB017 (peginterferon beta-1a) serve most often?
"BIIB017 is most often used as part of a parenteral drug administration therapy regimen. It has also shown efficacy in treating multiple sclerosis, carcinoma in situ, and parenteral drug administration." - Anonymous Online Contributor
Unverified Answer
What is the efficacy of BIIB017 in other medical research?
"25 studies are currently underway to study the efficacy of BIIB017 (peginterferon beta-1a), 13 of which are in Phase 3. The trials for BIIB017 are largely based in Milwaukee, Wisconsin, but there are 1,555 locations across the globe conducting these experiments." - Anonymous Online Contributor
Unverified Answer