Date: 2014-10-23
Type of information: Treatment of the first patient
phase: 2a
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 CNS 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 a treatment for essential tremor.
Disease: essential tremor
Therapeutic area: CNS diseases - Neurological diseases
Country:
Trial
details: The Phase 2a trial is a double blind, proof-of-concept study of SAGE-547 in patients who have a diagnosis of essential tremor with symptoms clearly present in at least one upper limb. The trial is expected to enroll 24 adult patients with essential tremor for at least 2 years, a score of ≥ 2 for at least one upper limb maneuver as measured by The Essential Tremor Rating Scale (TETRAS) performance subscale, and who are not currently taking medication for their tremor or are on a stable dose of medication for at least 28 days prior to enrollment. This trial is designed to provide clear data regarding the safety and tolerability of SAGE-547 using ratings and mechanical measures of motor activity designed to rapidly yield unambiguous endpoints. The activity of SAGE-547 will be assessed via physician examination, transducer measurement of tremor amplitude and full TETRAS and TETRAS performance subscale measurements. Patients will be administered either SAGE-547 or placebo intravenously for 12 hours on Day 1 and Day 10 in a double blind cross-over manner. Patients will be monitored for up to 30 days following treatment.
Latest
news: * On October 23, 2014, SAGE Therapeutics announced dose initiation of the first patient in a single-center Phase 2a clinical trial of SAGE-547, an allosteric modulator of GABAA receptors, in patients with essential tremor. This trial is designed to evaluate the safety, tolerability, pharmacokinetics and activity of SAGE-547 in patients with essential tremor, a neurological disorder that causes involuntary, rhythmic shaking with no known cause. The trial is being conducted under a newly accepted Investigational New Drug filing.
