Contributions
Abstract: P321
Type: Poster
Abstract Category: Clinical aspects of MS - 3 Paediatric MS
Background: Teriflunomide is a once-daily oral immunomodulator approved for relapsing-remitting MS. Teriflunomide 14 mg has shown consistent efficacy vs placebo in clinical studies in adults. There are unmet needs in the treatment of paediatric patients with MS. Patients with paediatric onset of MS represent 2-5% of all MS cases and can present with higher relapse rates and greater lesion burden compared with adult-onset MS.
Objective: To describe the design and provide an enrolment update for the phase 3, double-blind, randomized, placebo-controlled TERIKIDS study (NCT02201108) and open-label extension evaluating efficacy, safety, and pharmacokinetics (PK) of teriflunomide in children with relapsing MS (RMS).
Methods: Target recruitment: 165 patients aged 10-17 years with RMS meeting McDonald (2010) and International Pediatric MS Study Group (2013) criteria at screening, with ≥1 or ≥2 relapse(s) in the prior 12 or 24 months, respectively. Patients are being randomized 2:1 to teriflunomide or placebo, with adjustment to 14-mg adult-equivalent dose determined in a blinded PK run-in phase. The double-blind period will last 96 weeks or until a patient experiences relapse or exceeds protocol defined limits for enlarged/new T2 lesions, at which point patients have the option of entering an open-label period in which they receive teriflunomide. Primary endpoint: time to first clinical relapse after randomization. Secondary endpoints: proportions of relapse-free patients at 24, 48, 72, and 96 weeks; MRI and cognitive outcomes; and PK. Safety and tolerability will be evaluated by adverse event reporting at each visit.
Results: As of 5/5/17, 109 patients were randomized, with demographics as follows: 72 (66.1%) female; mean (SD) age, 14.7 (2.1) years; mean (SD) time since symptom onset, 2.24 (2.00) years. Of these, the majority (75.2%) had not received any MS treatment in the prior 2 years. Median (range) baseline EDSS score was 1.5 (0-4) and mean (SD) number of relapses within the previous 1 and 2 years was 1.5 (0.7) and 2.2 (1.1), respectively. At baseline, mean (SD) number of T2 lesions was 50.1 (38.2), and 58 (53.2%) patients had ≥1 gadolinium-enhancing lesion.
Conclusions: Paediatric patients enrolled in TERIKIDS to date have high levels of baseline disease activity and a relatively short disease duration. TERIKIDS will provide insights into efficacy, safety, tolerability, and PK of teriflunomide in the rare paediatric population with RMS.
Disclosure: Study supported by Sanofi Genzyme.
TC: Consulting fees (Bayer, Novartis, Sanofi Genzyme); advisory boards (Novartis, Roche-Genentech, Sanofi Genzyme); research support (Biogen, Novartis, Serono, Verily).
MT: Research support (Novartis, Sanofi Genzyme).
BB: Consulting fees (Novartis); non-remunerated advisory input (Biogen Idec, EMD Serono, Novartis, Sanofi Genzyme, Teva Neuroscience).
KD: Consulting fees (Merck Serono, Servier).
AA: Consulting fees (EMD Serono, Genzyme, Rosch); contracted research (Bayer, Biogen Idec, EMD Serono, Rosch).
NS, YW and BA: Nothing to disclose.
MR, RD, MD and SJ: Employees of Sanofi Genzyme.
PT: Employee of Sanofi Genzyme, with ownership interest.
LK: Author´s institution (University Hospital Basel) has received in the last 3 years and used exclusively for research support: steering committee, advisory board and consultancy fees (Actelion, Addex, Bayer HealthCare, Biogen Idec, Biotica, Genzyme, Lilly, Merck, Mitsubishi, Novartis, Ono Pharma, Pfizer, Receptos, Sanofi, Santhera, Siemens, Teva, UCB, XenoPort); speaker fees (Bayer HealthCare, Biogen Idec, Merck, Novartis, Sanofi, Teva); support of educational activities (Bayer HealthCare, Biogen Idec, CSL Behring, Genzyme, Merck, Novartis, Sanofi, Teva); licence fees (Neurostatus Systems GmbH); grants (Bayer HealthCare, Biogen, Merck, Novartis, Roche, Swiss MS Society, the Swiss National Research Foundation, the European Union and Roche Research Foundations).
Abstract: P321
Type: Poster
Abstract Category: Clinical aspects of MS - 3 Paediatric MS
Background: Teriflunomide is a once-daily oral immunomodulator approved for relapsing-remitting MS. Teriflunomide 14 mg has shown consistent efficacy vs placebo in clinical studies in adults. There are unmet needs in the treatment of paediatric patients with MS. Patients with paediatric onset of MS represent 2-5% of all MS cases and can present with higher relapse rates and greater lesion burden compared with adult-onset MS.
Objective: To describe the design and provide an enrolment update for the phase 3, double-blind, randomized, placebo-controlled TERIKIDS study (NCT02201108) and open-label extension evaluating efficacy, safety, and pharmacokinetics (PK) of teriflunomide in children with relapsing MS (RMS).
Methods: Target recruitment: 165 patients aged 10-17 years with RMS meeting McDonald (2010) and International Pediatric MS Study Group (2013) criteria at screening, with ≥1 or ≥2 relapse(s) in the prior 12 or 24 months, respectively. Patients are being randomized 2:1 to teriflunomide or placebo, with adjustment to 14-mg adult-equivalent dose determined in a blinded PK run-in phase. The double-blind period will last 96 weeks or until a patient experiences relapse or exceeds protocol defined limits for enlarged/new T2 lesions, at which point patients have the option of entering an open-label period in which they receive teriflunomide. Primary endpoint: time to first clinical relapse after randomization. Secondary endpoints: proportions of relapse-free patients at 24, 48, 72, and 96 weeks; MRI and cognitive outcomes; and PK. Safety and tolerability will be evaluated by adverse event reporting at each visit.
Results: As of 5/5/17, 109 patients were randomized, with demographics as follows: 72 (66.1%) female; mean (SD) age, 14.7 (2.1) years; mean (SD) time since symptom onset, 2.24 (2.00) years. Of these, the majority (75.2%) had not received any MS treatment in the prior 2 years. Median (range) baseline EDSS score was 1.5 (0-4) and mean (SD) number of relapses within the previous 1 and 2 years was 1.5 (0.7) and 2.2 (1.1), respectively. At baseline, mean (SD) number of T2 lesions was 50.1 (38.2), and 58 (53.2%) patients had ≥1 gadolinium-enhancing lesion.
Conclusions: Paediatric patients enrolled in TERIKIDS to date have high levels of baseline disease activity and a relatively short disease duration. TERIKIDS will provide insights into efficacy, safety, tolerability, and PK of teriflunomide in the rare paediatric population with RMS.
Disclosure: Study supported by Sanofi Genzyme.
TC: Consulting fees (Bayer, Novartis, Sanofi Genzyme); advisory boards (Novartis, Roche-Genentech, Sanofi Genzyme); research support (Biogen, Novartis, Serono, Verily).
MT: Research support (Novartis, Sanofi Genzyme).
BB: Consulting fees (Novartis); non-remunerated advisory input (Biogen Idec, EMD Serono, Novartis, Sanofi Genzyme, Teva Neuroscience).
KD: Consulting fees (Merck Serono, Servier).
AA: Consulting fees (EMD Serono, Genzyme, Rosch); contracted research (Bayer, Biogen Idec, EMD Serono, Rosch).
NS, YW and BA: Nothing to disclose.
MR, RD, MD and SJ: Employees of Sanofi Genzyme.
PT: Employee of Sanofi Genzyme, with ownership interest.
LK: Author´s institution (University Hospital Basel) has received in the last 3 years and used exclusively for research support: steering committee, advisory board and consultancy fees (Actelion, Addex, Bayer HealthCare, Biogen Idec, Biotica, Genzyme, Lilly, Merck, Mitsubishi, Novartis, Ono Pharma, Pfizer, Receptos, Sanofi, Santhera, Siemens, Teva, UCB, XenoPort); speaker fees (Bayer HealthCare, Biogen Idec, Merck, Novartis, Sanofi, Teva); support of educational activities (Bayer HealthCare, Biogen Idec, CSL Behring, Genzyme, Merck, Novartis, Sanofi, Teva); licence fees (Neurostatus Systems GmbH); grants (Bayer HealthCare, Biogen, Merck, Novartis, Roche, Swiss MS Society, the Swiss National Research Foundation, the European Union and Roche Research Foundations).