A Phase 3b/4 Long-Term Study of Alemtuzumab in Patients With Relapsing-Remitting Multiple Sclerosis: TOPAZ Study Design (P7.219)

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OBJECTIVE: To describe the design of A Long-Term Follow-up Study for Multiple Sclerosis Patients Who Have Completed the Alemtuzumab Extension Study (TOPAZ; NCT02255656). BACKGROUND: The phase 2 and 3 studies of alemtuzumab in patients with relapsing-remitting multiple sclerosis (RRMS; NCT00050778; NCT00530348; NCT00548405) showed that alemtuzumab had superior efficacy versus subcutaneous interferon-1a, with manageable safety. An extension study (NCT00930553) showed durable efficacy with no new safety signals at 4-year (phase 3) or 5-year (phase 2) follow-up. Longer-term data on a large patient population are currently lacking. DESIGN/METHODS: TOPAZ is a phase 3b/4, 5-year, observational, multicenter, multinational, open-label study in patients who have completed 蠅4 years of the extension study. Approximately 1200 patients are eligible; all have previously received alemtuzumab. Retreatment with alemtuzumab or other disease-modifying therapies (DMTs) is permitted. Alemtuzumab retreatment beyond the initial 2 courses will be administered on an as-needed basis at the discretion of the study investigator, consisting of alemtuzumab 12 mg/day intravenous on 3 consecutive days 蠅12 months after the most recent course. The subset of patients who receive alemtuzumab retreatment during TOPAZ will undergo regular safety monitoring for autoimmune adverse events (AEs), and annual human papilloma virus screening (women only), for 48 months after the last alemtuzumab dose, consistent with the risk management plan. The primary objective is to evaluate long-term safety (AEs, serious AEs, AEs of special interest [autoimmune AEs, infusion-associated reactions, malignancies, infections]). Secondary objectives include assessment of long-term efficacy (relapses, disability, and magnetic resonance imaging parameters), patient-reported quality-of-life outcomes, health resource utilization, and evaluation of alemtuzumab retreatment and treatment with other DMTs after alemtuzumab. RESULTS: Enrollment is expected to begin in November 2014. CONCLUSIONS: TOPAZ will provide extended clinical knowledge of alemtuzumab longer-term use, including the retreatment experience and treatment sequencing in patients with RRMS Study Supported by: Genzyme, a Sanofi company

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