Clinical trial NCT02978781: A Study to Evaluate SAGE-217 in Participants With Essential Tremor
Official title: A Phase 2a, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study Evaluating the Efficacy, Safety, Tolerability, and Pharmacokinetics of SAGE-217 in the Treatment of Subjects With Essential Tremor
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Essential Tremor
Keywords: Tremor
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2016-12-01
Enrollment: 34
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is a three-part, multicenter, Phase 2a study to evaluate the efficacy, safety, tolerability, and pharmacokinetics of SAGE-217 in adult participants with essential tremor.

Detailed description: Part A of the study was an open-label design with morning dosing of SAGE-217 for 7 days and included 16 participants, 8 of whom qualified for, and entered, Part B.

Part B had a double-blind, placebo-controlled, randomized withdrawal design with morning dosing for 7 days.

Part C was an open-label design with morning and evening dosing for 14 days and included a different set of 18 participants.

Parts A and B were stopped early (in advance of the planned sample size).

This study was previously posted by Sage Therapeutics. In November 2023, sponsorship of the trial was transferred to Biogen.

Eligibility criteria: Key Inclusion Criteria:

* Participant must have a diagnosis of Essential Tremor (ET), defined as bilateral postural tremor and kinetic tremor, involving hands and forearms, that is visible and persistent and the duration is \>5 years prior to screening.

Key Exclusion Criteria:

* Participant has presence of abnormal neurological signs other than tremor or Froment's sign.
* Participant has presence of known causes of enhanced physiological tremor.
* Participant has concurrent or recent exposure (14 days prior to admission visit) to tremorogenic drugs.
* Participant has had direct or indirect trauma to the nervous system within 3 months before the onset of tremor.
* Participant has historical or clinical evidence of tremor with psychogenic origin.
* Participant has convincing evidence of sudden tremor onset or evidence of stepwise deterioration of tremor.

Primary outcomes:
measure: Part A: Change From Baseline in Accelerometer-based Kinesia Kinetic Tremor Combined Score at Day 7
timeFrame: Baseline (Day 1) and Day 7 (8 hours postdose)
measure: Part B: Change From Randomization in Accelerometer-based Kinesia Kinetic Tremor Combined Score at Day 14
timeFrame: Randomization (Day 8, predose) and Day 14 (predose)
measure: Part C: Change From Baseline in the Accelerometer-based Kinesia Upper Limb Tremor Combined Score at Day 15
timeFrame: Baseline (Day 1) and Day 15
Secondary outcomes:
measure: Part A: Change From Baseline in Kinesia Upper Limb Total Score at Day 7
timeFrame: Baseline (Day 1) and Day 7 (8 hours postdose)
measure: Part A: Change From Baseline in Kinesia Upper Limb Individual Item Score at Day 7
timeFrame: Baseline (Day 1) and Day 7 (8 hours [hr] postdose [pd])
measure: Part A: Change From Baseline in the Tremor Research Group (TRG) Essential Tremor Rating Assessment Scale (TETRAS) Upper Limb Total Score at Day 7
timeFrame: Baseline (Day 1) and Day 7 (8 hours postdose)
measure: Change From Baseline in the TETRAS Upper Limb Individual Items (Performance Subscale Items 4a, 4b, or 4c) Scores at Day 7
timeFrame: Baseline (Day 1) and Day 7 (8 hours postdose)
measure: Part A: Change From Baseline in TETRAS Performance Subscale Score (Items 6, 7, and 8) at Day 7
timeFrame: Baseline (Day 1) and Day 7 (predose)
measure: Part B: Change From Randomization in the Kinesia Upper Limb Total Score at Day 14
timeFrame: Randomization (Day 8, predose) and Day 14 (predose)
measure: Part B: Change From Randomization in the Kinesia Upper Limb Individual Item Score at Day 14
timeFrame: Randomization (Day 8, predose) and Day 14 (predose)
measure: Part B: Change From Randomization in the TETRAS Upper Limb Total Score at Day 14
timeFrame: Randomization (Day 8, predose) and Day 14 (predose)
measure: Part B: Change From Randomization in the TETRAS Upper Limb Individual Item (Performance Subscale Items 4a and 4b) Score at Day 14
timeFrame: Randomization (Day 8, predose) and Day 14 (predose)
measure: Part B: Change From Randomization in the TETRAS Performance Subscale Item 4c (Kinetic Tremor) Combined Score at Day 14
timeFrame: Randomization (Day 8, predose) and Day 14 (predose)
measure: Part B: Change From Baseline in TETRAS Performance Subscale (Items 6, 7, and 8) Score at Day 14
timeFrame: Baseline (Day 1) and Day 14 (predose)
measure: Part C: Change From Baseline in the Kinesia Upper Limb Individual Item Score at Day 15
timeFrame: Baseline (Day 1) and Day 15
measure: Part C: Change From Baseline in the TETRAS Upper Limb Total Score at Day 15
timeFrame: Baseline (Day 1) and Day 15
measure: Part C: Change From Baseline in the TETRAS Upper Limb Individual Item (Performance Subscale Items 4a and 4b) Score at Day 15
timeFrame: Baseline (Day 1) and Day 15
measure: Part C: Change From Baseline in TETRAS Performance Subscale (Items 6, 7, and 8) Scores at Day 15
timeFrame: Baseline (Day 1) and Day 15
measure: Parts A, B and C: Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) or Serious Adverse Event (SAE)
timeFrame: From first dose of study drug through 14 days after the last dose (Up to 28 days)
measure: Part A, B and C: Number of Participants With Suicidal Ideation as Assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS) Score
timeFrame: Baseline and post-baseline (up to 28 days)
measure: Number of Participants With Clinically Significant Vital Signs
timeFrame: Up to 28 days
measure: Number of Participants With Clinically Significant Laboratory Parameters
timeFrame: Up to 28 days
measure: Number of Participants With Clinically Significant Electrocardiogram (ECG) Values
timeFrame: Up to 28 days
measure: Parts A and B: Change From Baseline in Stanford Sleepiness Scale (SSS)
timeFrame: Baseline (Day 1), Day 7 (predose) for Part A, Day 14 (predose) for Part B
measure: Parts A, B and C: Change From Baseline in Bond-Lader Visual Analogue Scale (VAS) Score
timeFrame: Baseline (Day 1), Day 7 (predose) for Part A, Day 14 (predose) for Part B and Day 15 for Part C
measure: Parts A, B and C: Participant's Feeling After Taking the Study Drug as Assessed by Drug Effects Questionnaire (DEQ-5) Score
timeFrame: Baseline (Day 1), Day 7 (2 hours postdose) for Part A, Day 14 (2 hours postdose) for Part B and Day 15 for Part C
Other outcomes:
Locations:
city: Anniston
country: United States
facility: Sage Investigational Site
state: Alabama
city: Little Rock
country: United States
facility: Sage Investigational Site
state: Arkansas
city: Rogers
country: United States
facility: Sage Investigational Site
state: Arkansas
city: Cerritos
country: United States
facility: Sage Investigational Site
state: California
city: Fountain Valley
country: United States
facility: Sage Investigational Site
state: California
city: Oceanside
country: United States
facility: Sage Investigational Site
state: California
city: San Diego
country: United States
facility: Sage Investigational Site
state: California
city: Englewood
country: United States
facility: Sage Investigational Site
state: Colorado
city: Clearwater
country: United States
facility: Sage Investigational Site
state: Florida
city: DeLand
country: United States
facility: Sage Investigational Site
state: Florida
city: Lauderdale Lakes
country: United States
facility: Sage Investigational Site
state: Florida
city: Orlando
country: United States
facility: Sage Investigational Site
state: Florida
city: Ormond Beach
country: United States
facility: Sage Investigational Site
state: Florida
city: Saint Petersburg
country: United States
facility: Sage Investigational Site
state: Florida
city: Tampa
country: United States
facility: Sage Investigational Site
state: Florida
city: Atlanta
country: United States
facility: Sage Investigational Site
state: Georgia
city: Decatur
country: United States
facility: Sage Investigational Site
state: Georgia
city: Chicago
country: United States
facility: Sage Investigational Site
state: Illinois
city: Urbana
country: United States
facility: Sage Investigational Site
state: Illinois
city: Saint Louis
country: United States
facility: Sage Investigational Site
state: Missouri
city: Springfield
country: United States
facility: Sage Investigational Site
state: Missouri
city: Raleigh
country: United States
facility: Sage Investigational Site
state: North Carolina
city: Cincinnati
country: United States
facility: Sage Investigational Site
state: Ohio
city: Dayton
country: United States
facility: Sage Investigational Site
state: Ohio
city: Nashville
country: United States
facility: Sage Investigational Site
state: Tennessee
city: Fort Worth
country: United States
facility: Sage Investigational Site
state: Texas
Study officials:
References: