Evaluate SAGE-547 in Participants With Essential Tremor

Sage Therapeutics

Status and phase: Completed; Phase 2

Conditions: Essential Tremor

Treatments: Drug: Placebo; Drug: SAGE-547

Study type: Interventional

Funder types: Industry

Identifiers

NCT02277106

547-ETD-201

Details and patient eligibility

About

Stage 1 is a double-blind, proof-of-concept study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of SAGE-547 Injection in male and female participants with essential tremor in the upper limb.

Stage 2 is an open-label arm designed to evaluate the safety, tolerability, PK, and effectiveness of SAGE-547 Injection at a higher dose than in Stage 1. Participants who completed Stage 1 were invited to participate in Stage 2.

Enrollment: 25 patients

Sex: All

Ages: 35 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Males and females, 35-75 years old with a diagnosis of essential tremor with symptoms clearly present in at least 1 upper limb; participant has had tremor present for at least 2 years prior to Screening
• Off medication, or on a stable dose of medication for their tremor for at least 28 days prior to Screening

Exclusion criteria

• Recent history or active clinically significant manifestations of metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, musculoskeletal, dermatological, urogenital, eyes, ears, nose, or throat, psychiatric, or neurological (other than essential tremor) disorders
• Medical history of seizures

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

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25 participants in 3 patient groups

Stage 1 (Double-blind): SAGE 547, Then Placebo

Experimental group

Description:

Participants received a 12-hour intravenous (IV) infusion of SAGE 547, at ascending doses of 29, 58, and 86 micrograms per kilogram of body weight per hour (μg/kg/h), 4 hours each, on Day 1 of Stage 1 \[Treatment Period 1 (TP 1)\]. After a washout period of approximately 7 days, participants received a 12-hour IV infusion of SAGE-547 matching-placebo, on Day 10 of Stage 1 \[Treatment Period 2 (TP 2)\].

Treatment:

Drug: Placebo

Drug: SAGE-547

Stage 1 (Double-blind): Placebo, Then SAGE-547

Experimental group

Description:

Participants received a 12-hour IV infusion of SAGE-547 matching-placebo on Day 1 of Stage 1 (TP 1). After a washout period of approximately 7 days, participants received a 12-hour IV infusion of SAGE 547 at ascending doses of 29, 58, and 86 μg/kg/h, 4 hours each, on Day 10 of Stage 1 (TP 2).

Treatment:

Drug: Placebo

Drug: SAGE-547

Stage 2 (Open Label): SAGE-547

Experimental group

Description:

Participants who completed Stage 1 were invited to receive a 10-hour IV infusion of SAGE 547, at ascending doses of 90 μg/kg/h for 1 hour, 120 μg/kg/h for 1 hour, and 150 μg/kg/h for 8 hours on Day 1 of Stage 2.

Treatment:

Drug: SAGE-547