A Study to Evaluate SAGE-217 in Participants With Essential Tremor

Biogen

Status and phase: Completed; Phase 2

Conditions: Essential Tremor

Treatments: Drug: Placebo; Drug: SAGE-217

Study type: Interventional

Funder types: Industry

Identifiers

NCT02978781

217-ETD-201

Details and patient eligibility

About

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.

Full 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.

Enrollment: 34 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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34 participants in 5 patient groups, including a placebo group

Part A: SAGE-217 Oral Solution

Experimental group

Description:

Participants received SAGE-217 10 mg oral solution on Day 1, 20 mg on Day 2 and 30 mg on Days 3 to 7 with food in the morning.

Treatment:

Drug: SAGE-217

Drug: SAGE-217

Part A: SAGE-217 Capsules

Experimental group

Description:

Participants received SAGE-217 10 mg capsules on Day 1, 20 mg on Day 2 and 30 mg on Days 3 to 7, orally, with food in the morning.

Treatment:

Drug: SAGE-217

Drug: SAGE-217

Part B: Placebo

Placebo Comparator group

Description:

Participants who received maximum tolerated dose of SAGE-217 in Part A and achieved response on Day 8 were randomized to receive to SAGE-217 matching placebo for 7 days beginning on Day 8 with food in the morning.

Treatment:

Drug: Placebo

Part B: SAGE-217 Capsules

Experimental group

Description:

Participants who received maximum tolerated dose of SAGE-217 in Part A and achieved response on Day 8 were randomized to receive to SAGE-217 for 7 days beginning on Day 8 with food in the morning.

Treatment:

Drug: SAGE-217

Drug: SAGE-217

Part C: SAGE-217 Capsules

Experimental group

Description:

Participants received SAGE-217 10 mg capsules on Day 1, 20 mg on Day 2, 30 mg on Day 3, orally, with food in the evening. Beginning on Day 4 through Day 14, participants received a 40-mg total daily dose (administered as 10 mg with food in the morning and 30 mg with food in the evening).

Treatment:

Drug: SAGE-217

Drug: SAGE-217