A two year, randomized, placebo-controlled study to evaluate efficacy, safety, tolerability, and pharmacokinetics of teriflunomide once daily in pediatric patients with relapsing forms of multiple sclerosis

• Register: EUCTR
• Last refreshed on: 14 October 2024
• Main ID: EUCTR2011-005249-12-FR
• Date of registration: 17/06/2015
• Prospective Registration: Yes
• Primary sponsor: Genzyme Corporation
• Public title: A two year, randomized, placebo-controlled study to evaluate efficacy, safety, tolerability, and pharmacokinetics of teriflunomide once daily in pediatric patients with relapsing forms of multiple sclerosis
• Scientific title: 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 - TERIKIDS
• Target sample size: 165
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-005249-12
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: yes Other trial design description: Option to go in an open label teriflunomide arm for patients having a relapse after PK run-in phase If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Belgium, Bulgaria, Canada, China, Estonia, France, Greece, Ireland, Israel, Italy, Lebanon, Lithuania, Netherlands, Poland, Portugal, Russian Federation, Slovenia, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Direction des Opérations Cliniques
• Affiliation: sanofi-aventis France

• Name: Direction des Opérations Cliniques
• Affiliation: sanofi-aventis France

Key inclusion & exclusion criteria

Inclusion criteria:
- Patients with relapsing multiple sclerosis (MS) based on McDonald criteria of 2010 and International Pediatric Multiple Sclerosis Study Group (IPMSG) criteria for pediatric MS:
-at least one relapse in the 12 months preceding randomization, or
-at least 2 relapses in the 24 months preceding randomization.
- Equal or less than 17 years of age and equal or greater than 10 years of age at randomization.
- Signed informed consent/assent obtained from patient and patient's legal representative.

Are the trial subjects under 18? yes
Number of subjects for this age range: 165
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Expanded disability status scale (EDSS) score greater than 5.5 at screening or randomization visit.
- Relapse within 30 days prior to randomization.
- Treated with cladribine or mitoxantrone within 2 years preceding randomization.
- Treated with glatiramer acetate, interferons, natalizumab, introvenous immunoglobulins or other immunosuppressant or immunomodulatory agents such as cyclophosphamide, azathioprine, cyclosporine, methotrexate, mycophenolate, dimetthyl fumrate, fingolimod less than 3 months or five half-lives prior to randomization, whichever is greater.
- History of human immunodeficiency virus (HIV) infection.
- Contraindication for magnetic resonance imaging (MRI).
- Pregnant or breast-feeding females or those who plan to become pregnant during the study.
- Female patients of child-bearing potential not using highly effective contraceptives or (double barrier) contraceptive method.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Multiple Sclerosis
MedDRA version: 18.0 Level: PT Classification code 10028245 Term: Multiple sclerosis System Organ Class: 10029205 - Nervous system disorders

Intervention(s)

Product Name: Teriflunomide
Product Code: HMR1726
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TERIFLUNOMIDE
CAS Number: 108605-62-5
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 14-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Product Name: Teriflunomide
Product Code: HMR1726
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TERIFLUNOMIDE
CAS Number: 108605-62-5
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 7-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Product Name: Teriflunomide
Product Code: HMR1726
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TERIFLUNOMIDE
CAS Number: 108605-62-5
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 3.5-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Over 96 weeks

Secondary Objective: To assess the effect of teriflunomide in comparison to placebo on disease activity/progression measured by brain MRI and on cognitive function.
To evaluate the safety and tolerability of teriflunomide in comparison to placebo.
To evaluate the pharmacokinetics (PK) of teriflunomide.

Primary end point(s): Time to first clinical relapse after randomization

Main 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.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1 and 9 : Over 96 weeks
2, 3, 4, 5, 6 and 7 : At randomization, at 24, 48 and 96 weeks
8 : At randomization, then every 24 weeks (SDMT only) and at 96 weeks
10 : A total of 3 PK samples (blood samples) will be collected during the beginning of study, i.e., the 4-week PK run-in period (weeks 2, 3 and 4); patients entering the open label teriflunomide arm will re-do PK run-in (3 blood samples)

Secondary end point(s): 1 - Proportion of relapse free patients
2 - Number of of new/newly enlarged T2 lesions
3 - Number of T1 Gd-enhancing T1 lesions
4 - Change in volume of T2 lesions
5 - Change in volume of T1 hypointense lesions
6 - Number of new T1 hypointense lesions
7 - Change in brain atrophy
8 - Change in performance on symbol digit modalities test (SDMT) and Cognitive Battery Test
9 - Overview of safety including adverse events (AEs) based on AE reporting, physical examinations, vital signs, clinical laboratories, suspected neuropathy, electrocardiography (ECG), suspected infection
10 - Teriflunomide pharmacokinetics (PK) to allow the dose adjustment to the 14 mg adult equivalent dose for the rest of the
study

Secondary ID(s)

2011-005249-12-EE

EFC11759

Source(s) of Monetary Support

Genzyme Corporation

Results

• Results available: Yes
• Date Posted: 29/01/2021
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-005249-12/results