Date: 2015-08-17
Type of information: Treatment of the first patient
phase: 3
Announcement: treatment of the first patient
Company: Sage Therapeutics (USA - MA)
Product: SAGE-547
Action
mechanism: Allosteric modulator. SAGE-547 is an allosteric modulator of both synaptic and extra-synaptic GABAA receptors. GABAA receptors are widely regarded as validated drug targets for a variety of disorders, with decades of research and multiple approved drugs targeting these receptor systems. SAGE-547 is an intravenous agent in Phase 1/2 clinical development as an adjunctive therapy, a therapy combined with current therapeutic approaches, for the treatment of super-refractory status epilepticus (SRSE), as well as in exploratory Phase 2 clinical trials for the treatment of essential tremor and as an adjunctive therapy for the treatment of severe postpartum depression (PPD). In 2014, the FDA granted both Fast Track and orphan drug designation to SAGE-547 for the treatment of SRSE.
Disease: super-refractory status epilepticus (SRSE)
Therapeutic area: CNS diseases - Neurological diseases
Country: USA
Trial
details: This randomized, double-blind, placebo-controlled trial has been designed to evaluate the efficacy and safety of SAGE-547 administered as a continuous intravenous infusion to subjects in Super-Refractory Status Epilepticus (SRSE). (NCT02477618)
Latest
news: * On August 17, 2015, Sage Therapeutics announced it has treated the first patient enrolled in the STATUS Trial (SAGE-547 Treatment as Adjunctive Therapy Utilized in Status Epilepticus), a global, Phase 3, randomized, double-blind, placebo-controlled clinical trial to evaluate SAGE-547 as a treatment for patients with super-refractory status epilepticus (SRSE).The STATUS Trial is designed to assess the efficacy and safety of SAGE-547 in approximately 126 patients with SRSE, aged two years or older, and will be conducted in the U.S., Canada and Europe. Patients will be randomized 1:1 to receive either SAGE-547 or placebo in addition to standard-of-care third-line anti-seizure agents for six days. The planned primary endpoint of the Phase 3 clinical trial will be successful resolution of status epilepticus (SE) after weaning the patient off all third-line agents, and SAGE-547 or placebo, without resumption of SE within 24 hours after completion of blinded SAGE-547 or placebo administration.SAGE recently announced that agreement has been reached with the FDA under a Special Protocol Assessment on the trial design, endpoints and statistical approach of the Phase 3 clinical trial. The SPA provides agreement from the FDA that the Phase 3 STATUS Trial can adequately address objectives in support of a U.S. regulatory submission for approval of SAGE-547 for the treatment of patients with SRSE. SAGE\'s Phase 3 open-label expanded access protocol, designated Study 302, was initiated in April 2015 and continues its enrollment. Study 302 is designed to make SAGE-547 available to patients in the U.S. who are affected by SRSE and who have not been admitted to, nor can be transferred to, a planned STATUS Trial clinical site. * On August 6, 2015, Sage Therapeutics announced it has reached agreement with the FDA under a Special Protocol Assessment (SPA) for the STATUS Trial. The SPA provides agreement from the FDA that the Phase 3 STATUS Trial can adequately address objectives in support of a U.S. regulatory submission for approval of SAGE-547 for the treatment of patients with SRSE.
In a completed Phase 1/2 open-label clinical trial, SAGE-547 demonstrated robust activity, with a 77 percent response rate in 22 evaluable patients with SRSE, and also a favorable tolerability profile. Independent of treatment response, six patient deaths occurred within the trial period, all driven by underlying conditions. Although 64 percent of patients reported serious adverse events, none were drug-related as determined by the Safety Review Committee.
