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

Conditions: Multiple Sclerosis
MedDRA version: 18.0Level: PTClassification code 10028245Term: Multiple sclerosisSystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2011-005249-12-PL

Lead Sponsor: Genzyme Corporation

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 165

Inclusion Criteria

-Patients with relapsing multiple sclerosis are eligible. Patients should meet the criteria of MS based on McDonald criteria 2010 and International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria for pediatric MS, version of 2012 (5) and have:
- at least one relapse (or attack) in the 12 months preceding randomization or
- at least two relapses (or attack) in the 24 months preceding randomization
-=17 years of age and =10 years of age at randomization
-Signed informed consent/assent obtained from patient and patient's legal representative (parents or guardians) according to local regulations.
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

• EDSS score > 5.5 at screening or randomization visits
• Relapse within 30 days prior to randomization
• Treated with
- glatiramer acetate, interferons, or dimethyl fumarate within 1 month prior to randomization
- fingolimod, or intravenous immunoglobulins within 3 months prior to randomization
- natalizumab, other immunosuppressant or immunomodulatory agents such as cyclophosphamide, azathioprine, cyclosporine, methotrexate, mycophenolate, within 6 months prior to randomization
- cladribine or mitoxantrone within 2 years prior to randomization
• Treated with alemtuzumab at any time
• History of HIV infection
• Contraindication for 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 contraceptive method (contraception in both female and male is required).

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary end point(s): Time to first clinical relapse after randomization<br>;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.<br>;Secondary Objective: To assess the effect of teriflunomide in comparison to placebo on disease activity/progression measured by brain MRI and on cognitive function.<br>To evaluate the safety and tolerability of teriflunomide in comparison to placebo.<br>To evaluate the pharmacokinetics (PK) of teriflunomide.<br>;Timepoint(s) of evaluation of this end point: Over 96 weeks<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 3 - Number of new/newly enlarged T2 lesions<br>4 - Number of T1 Gd-enhancing T1 lesions<br>5 - Change in volume of T2 lesions<br>- Change in volume of T1 hypointense lesions<br>6 - Number of new T1 hypointense lesions<br>7 - Proportion of patients free of new or enlarged MRI T2-lesions<br>8 - Brain atrophy<br>9 - Change in performance on symbol digit modalities test (SDMT) and Cognitive Battery Test<br>10 - Safety, as assessed by clinical, laboratory, ECG, and vital signs events<br>11 - Teriflunomide pharmacokinetics (PK);Timepoint(s) of evaluation of this end point: 2: at 24, 48, 72 and 96 weeks<br>3, 4, 5, 6, 8 and 10: over 96 weeks<br>7: at 48 weeks and 96 weeks<br>9: at randomization, then every 24 weeks (SDMT only) and at 96 weeks<br>11: at Weeks 2, 3, 4, 8, 12, 24, 36 and EOT

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