NCT02201108

Efficacy, Safety and Pharmacokinetics of Teriflunomide in Pediatric Patients With Relapsing Forms of Multiple Sclerosis

A Two Year, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial to Evaluate Efficacy, Safety, Tolerability, and Pharmacokinetics of Teriflunomide Administered Orally Once Daily in Pediatric Patients With Relapsing Forms of Multiple Sclerosis Followed by an Open-Label Extension

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 166 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 10 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To assess the effect of teriflunomide in comparison to placebo on disease activity measured by time to first clinical relapse after randomization in children and adolescents 10 to 17 years of age with relapsing forms of multiple sclerosis (MS). Secondary Objective: * To assess the effect of teriflunomide in comparison to placebo on disease activity/progression measured by brain magnetic resonance imaging (MRI) and on cognitive function. * To evaluate the safety and tolerability of teriflunomide in comparison to placebo. * To evaluate the pharmacokinetics (PK) of teriflunomide.

Detailed description

The study duration included a screening period up to 4 weeks, a double-blind treatment period of up to 96 weeks, an open-label period which included the remainder of the initial 96 weeks, where applicable, and a 96-week extension, i.e., up to a maximum of 192 weeks after randomization. There was a follow-up period of 4 weeks for participants discontinuing treatment. Within the 96 weeks double-blind treatment period, the first 4 weeks were PK run-in phase in which PK samples (blood samples) were collected from participants and then 4 weeks of analysis (no samples drawn). The PK run-in phase (total 8 weeks) was intended to provide individual PK parameters to allow the dose adjustment to the 14 milligrams (mg) adult-equivalent dose for the rest of the study. Participants who experienced a relapse after the PK run-in phase (8 weeks) and confirmed by the Relapse Adjudication Panel and participants who fulfilled MRI criteria (high number of new lesions at weeks 36, 48 or 72 compared to previous images) had the option to continue in an open-label teriflunomide treatment arm up to 192 weeks from randomization. An optional additional extension period was available for young participants with teriflunomide until the participants are 18 years old and/or able to switch to commercial product, whichever comes first.

Conditions

• Multiple Sclerosis

Interventions

Type	Name	Description
DRUG	Teriflunomide	Pharmaceutical form:film-coated tablet, Route of administration: oral
DRUG	Placebo	Pharmaceutical form:tablet, Route of administration: oral

Timeline

• Start date: 2014-07-16
• Primary completion: 2019-10-25
• Completion: 2024-07-29
• First posted: 2014-07-25
• Last updated: 2025-02-06
• Results posted: 2020-11-30

Locations

57 sites across 22 countries: United States, Belgium, Bulgaria, Canada, China, Estonia, France, Greece, Israel, Lebanon, Lithuania, Morocco, Netherlands, North Macedonia, Portugal, Russia, Serbia, Spain, Tunisia, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study