Safety, Efficacy, and Tolerability of NBI-827104 for the Treatment of Essential Tremor

NCT04880616 | Completed Phase 2

• 2 other identifiers: NBI-827104-ET20162020-006012-24
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 1

Brief Summary

The main objective of this study is to evaluate the efficacy, safety, and tolerability of NBI-827104 in adults with essential tremor.

Conditions

Essential Tremor

Keywords

Movement disorder; Tremor syndrome

Outcome Measures

Primary Outcomes (1)

• Change from Baseline to Day 28 of Each Treatment Period in Amplitude at Peak Frequency of Postural Tremor

  Baseline and Day 28 of each treatment period

Secondary Outcomes (3)

• Change from Baseline in Essential Tremor Rating Assessment Scale (TETRAS) Performance Score

  Baseline up to Day 28 of each treatment period

• Change from Baseline in TETRAS Activities of Daily Living (ADL) Score

  Baseline up to Day 28 of each treatment period

• Clinical Global Impression of Change (CGI-C)

  Up to Day 28 of each treatment period

Study Arms (2)

Sequence 1

EXPERIMENTAL

In treatment period 1 participants will receive increasing doses of NBI-827104 for 28 days. After a 14-day washout period, participants will receive matching placebo for 28 days in treatment period 2.

Drug: NBI-827104; Drug: Placebo

Sequence 2

EXPERIMENTAL

In treatment period 1 participants will receive matching placebo for 28 days. After a 14-day washout period, participants will receive increasing doses of NBI-827104 for 28 days in treatment period 2.

Drug: NBI-827104; Drug: Placebo

Interventions

NBI-827104 DRUG

Capsules for oral administration

Sequence 1; Sequence 2

Placebo DRUG

Capsules matching NBI-827104 for oral administration

Sequence 1; Sequence 2

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subjects, 18 to 75 years of age, inclusive at screening.
• Body mass index (BMI) between 18 and 35 kg/m², inclusive, at screening.
• Diagnosis of Essential Tremor (inclusive of Essential Tremor plus) as defined by the Movement Disorders Society Consensus Criteria for Tremor.
• Confirmation of bilateral upper limb action tremor in the absence of overt dystonia, ataxia, or parkinsonism.
• History of onset of tremor before 65 years of age.
• Tremor Performance score of ≥ 2 on at least 2 of the 6 upper limb manoeuvres (Item 4) on the TETRAS Performance Subscale and a total TETRAS Performance score ≥15 at screening.
• All women of childbearing potential and all males must practice effective contraception during the study and be willing and able to continue contraception for at least 90 days after their last dose of study drug.

You may not qualify if:

• Evidence of any acute (at screening or prior to first dose) or chronic disease or condition that could interfere with, or for which the treatment might interfere with, the conduct of the study, or that would pose an unacceptable risk to the subject in the opinion of the investigator.
• Have direct or indirect trauma to the nervous system within 3 months preceding the onset of tremor.
• Have known history of other medical or neurological conditions that may cause or explain subject's tremor.
• Have had prior magnetic resonance guided focused ultrasound or surgical intervention (e.g., deep brain stimulation, ablative thalamotomy or gamma knife thalamotomy).
• Have a history or evidence of clinically significant cardiac conduction abnormality, uncontrolled bradyarrhythmia, heart failure, or long QT syndrome.

Sponsors & Collaborators

• Neurocrine Biosciences: lead

Study Sites (1)

Neurocrine Clinical Site

Leiden, CL, 2333, Netherlands

Location

MeSH Terms

Conditions

Essential Tremor; Movement Disorders

Condition Hierarchy (Ancestors)

Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Clinical Development Lead

  Neurocrine Biosciences

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2021
• First Posted: May 11, 2021
• Study Start: April 20, 2021
• Primary Completion: May 25, 2022
• Study Completion: June 8, 2022
• Last Updated: July 12, 2022

Record last verified: 2022-07

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1)