Study to Evaluate the Efficacy and Safety of Dimethyl Fumarate (Tecfidera) and Peginterferon Beta-1a (Plegridy) for the Treatment of Relapsing-Remitting Multiple Sclerosis in Pediatric Participants

NCT03870763 | Terminated Phase 3 Results DMC FDA Drug

• 2 other identifiers: 800MS3012018-000516-22
• Study Type: interventional
• Target: 11
• Locations: 12 countries
• Sites: 19

Brief Summary

The main objective of the study is to evaluate the efficacy of dimethyl fumarate (Tecfidera) and peginterferon beta-1a (Plegridy), both compared with placebo, in pediatric participants with RRMS. The other objectives of this study are to evaluate the safety and tolerability of dimethyl fumarate and peginterferon beta-1a and to assess the effect of dimethyl fumarate and peginterferon beta-1a, both compared with placebo, on additional clinical and radiological measures of disease activity.

Conditions

Multiple Sclerosis, Relapsing-Remitting

Keywords

Pediatric, Multiple Sclerosis

Outcome Measures

Primary Outcomes (1)

• Time to First Relapse

  A clinical relapse is defined as new or recurrent neurological symptoms, not associated with fever, lasting for at least 24 hours, and followed by a period of 30 days of stability or improvement. Time to relapse is defined as from the first dose of the study drug to the day of relapse. Time to First Relapse is estimated by Kaplan Mayer method.

  Baseline up to Week 96

Secondary Outcomes (4)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Baseline up to Week 100

• Number of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans at Weeks 48 and 96

  Weeks 48 and 96

• Number of Galdolinium (Gd)-Enhancing Lesions at Weeks 48 and 96

  Weeks 48 and 96

• Annualized Relapse Rate

  Up to Week 96

Study Arms (3)

Dimethyl Fumarate 240 mg

EXPERIMENTAL

Participants will receive dimethyl fumarate 240 milligrams (mg) capsule twice daily (BID) orally and placebo subcutaneous (SC) injection every 2 weeks for up to 96 weeks (2 years).

Drug: Dimethyl Fumarate

Peginterferon Beta-1a 125 µg

EXPERIMENTAL

Participants will receive peginterferon beta-1a 125 micrograms (µg) SC injection every 2 weeks and placebo capsule BID orally for up to 96 weeks (2 years).

Drug: Peginterferon Beta-1a

Placebo

PLACEBO COMPARATOR

Participants will receive placebo SC injection every 2 weeks and placebo capsule BID orally for up to 96 weeks (2 years)

Drug: Placebo

Interventions

Dimethyl Fumarate DRUG

Administered as specified in the treatment arm.

Also known as: Tecfidera, BG00012

Dimethyl Fumarate 240 mg

Peginterferon Beta-1a DRUG

Administered as specified in the treatment arm.

Also known as: Plegridy, BIIB017

Peginterferon Beta-1a 125 µg

Placebo DRUG

Administered as specified in the treatment arm.

Placebo

Eligibility Criteria

• Age: 10 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 5.0.
• Must have a body weight of ≥30 kg
• Must have experienced ≥1 relapse in the 12 months prior to randomization (Day 1), or must have evidence of asymptomatic disease activity seen on MRI in the 6 months prior to randomization, or ≥2 relapses in the 24 months prior to randomization (Day 1). Relapse is defined as the occurrence of a clinical demyelination event regardless of whether the event is a first or subsequent demyelinating event.

You may not qualify if:

• Participants having primary progressive, secondary progressive, or progressive RMS.
• Disorders mimicking MS, such as other demyelinating disorders, systemic autoimmune disorders, metabolic disorders, and infectious disorders.
• History of clinically significant cardiovascular, pulmonary, GI, hepatic, renal, endocrinologic, hematologic, immunologic, metabolic, dermatologic, growth, developmental, psychiatric (including depression), neurologic (other than MS), and/or other major disease and/or laboratory abnormality indicative thereof, that would preclude participation in a clinical study
• Occurrence of an MS relapse within the 30 days prior to randomization (Day 1) and/or the subject has not stabilized from a previous relapse prior to randomization

Sponsors & Collaborators

• Biogen: lead

Study Sites (23)

Research Site

Raleigh, North Carolina, 27607, United States

Location

Research Site

Medellín, Colombia

Location

Research Site

Tallinn, 11315, Estonia

Location

Research Site

Budapest, 1083, Hungary

Location

Research Site

Ar Ramtha, Jordan

Location

Research Site

Petaling Jaya, Malaysia

Location

Research Site

Seberang Jaya, Malaysia

Location

Research Site

Guadalajara, Mexico

Location

Research Site

Morelia, Mexico

Location

Research Site

Santa Cruz, Mexico

Location

Research Site

Dammam, Saudi Arabia

Location

Research Site

Riyadh, Saudi Arabia

Location

Research Site

Seoul, 06351, South Korea

Location

Research Site

Taipei, 10002, Taiwan

Location

Research Site

Taoyuan District, 333, Taiwan

Location

Research Site

Bangkok, Thailand

Location

Research Site

Manouba, Tunisia

Location

Research Site

Monastir, Tunisia

Location

Research Site

Sfax, Tunisia

Location

Research Site

Tunis, Tunisia

Location

Research Site

Ankara, Turkey (Türkiye)

Location

Research Site

Izmir, Turkey (Türkiye)

Location

Research Site

Samsun, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Multiple Sclerosis, Relapsing-Remitting; Multiple Sclerosis

Interventions

Dimethyl Fumarate; peginterferon beta-1a

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Fumarates; Dicarboxylic Acids; Acids, Acyclic; Carboxylic Acids; Organic Chemicals

Limitations and Caveats

The study was terminated because of long-term difficulties in fulfilling the enrolment commitments and changes in paediatric MS landscape which no longer support placebo-controlled trials. Decision to stop study was not based on safety concerns.

Results Point of Contact

• Title: US Biogen Clinical Trial Center
• Organization: Biogen

clinicaltrials@biogen.com

866-633-4636

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 7, 2019
• First Posted: March 12, 2019
• Study Start: March 19, 2019
• Primary Completion: July 21, 2022
• Study Completion: July 21, 2022
• Last Updated: June 15, 2023
• Results First Posted: May 16, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will share

Locations

MY (2); TW (2); TH (1); ES (1); KR (1); TU (3); SA (2); JO (1); US (1); HU (1); CO (1); ME (3)