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

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

Official Title
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A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of NBI-827104 in Subjects With Essential Tremor

Conditions
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Essential Tremor

Intervention / Treatment
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* Drug: NBI-827104
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sequence 1<br>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<br>* Capsules for oral administration<br>Drug: Placebo<br>* Capsules matching NBI-827104 for oral administration<br>|
| Experimental: Sequence 2<br>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<br>* Capsules for oral administration<br>Drug: Placebo<br>* Capsules matching NBI-827104 for oral administration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Movement disorder, Tremor syndrome