Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated With HD

Study Overview
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Brief Summary
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To determine the efficacy of deutetrabenazine to control symptoms of dysphagia associated with HD.

Detailed Description
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The primary endpoint is the change in swallow function/dysphagia from baseline to maintenance therapy.

Official Title
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Efficacy of Deutetrabenazine in Huntington's Disease Patients With Dysphagia: A Randomised, Placebo-controlled Pilot Study

Conditions
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Huntington Disease

Intervention / Treatment
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* Drug: Deutetrabenazine Oral Tablet [Austedo]
* Drug: Placebo oral tablet


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed diagnosis of HD Symptoms of dysphagia Must be able to swallow tablets Exclusion Criteria: Other confounding diseases that affect swallowing Depression Hepatic impairment Renal impairment Dementia

Ages Eligible for Study
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Minimum Age: 21 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: Parallel Assignment
Interventional Model Description: Placebo-controlled pilot study
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Deutetrabenazine<br>The participants randomized to this group will receive oral deutetrabenazine for 8 weeks. Week 1: 6 mg once daily; Week 2: 6 mg twice daily (BID); Week 3: 9 mg BID; Week 4: 12 BID; Week 5: 15 mg BID; Week 6: 18 mg BID; Week 7: 21 mg BID; Week 8: 24 mg BID. | Drug: Deutetrabenazine Oral Tablet [Austedo]<br>* The participants randomized to this group will receive oral deutetrabenazine for 8 weeks. Week 1: 6 mg tab once daily; Week 2: 6 mg tab (BID); Week 3: 9 mg tab (BID); Week 4: 12 mg tab (BID); Week 5: 15 mg (6 mg tab + 9 mg tab BID); Week 6: 18 mg (9 mg tab + 9 mg tab BID); Week 7: 21 mg (9 mg tab + 12 mg tab BID); Week 8: 24 mg (12 mg tab BID).<br>* Other names: Austedo;|
| Placebo Comparator: Placebo<br>The participants randomized to this group will receive oral placebo for 8 weeks. Week 1: 6 mg once daily; Week 2: 6 mg twice daily (BID); Week 3: 9 mg BID; Week 4: 12 BID; Week 5: 15 mg BID; Week 6: 18 mg BID; Week 7: 21 mg BID; Week 8: 24 mg BID. | Drug: Placebo oral tablet<br>* The participants randomized to this group will receive oral placebo for 8 weeks. Week 1: 6 mg tab once daily; Week 2: 6 mg tab (BID); Week 3: 9 mg tab (BID); Week 4: 12 mg tab (BID); Week 5: 15 mg (6 mg tab + 9 mg tab BID); Week 6: 18 mg (9 mg tab + 9 mg tab BID); Week 7: 21 mg (9 mg tab + 12 mg tab BID); Week 8: 24 mg (12 mg tab BID).<br>* Other names: Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease. | The change in swallow function/dysphagia from baseline to maintenance therapy using the clinical swallow assessment Huntington's Disease Dysphagia Scale (HDDS). The Huntington's Disease Dysphagia Scale is an 11-item scale used to monitor swallowing difficulties in persons in early to advanced disease stages. The Huntington's Disease Dysphagia Scale includes questions related to the preparatory oral, oral, pharyngeal, and esophageal phases of swallow. The response options are the following: Question 1-4 and 6-11: 1 No, almost never; 2 Yes, seldom; 3 Yes, sometimes; 4 Yes, frequently; 5 Yes, almost always. Question 5: 1 Yes, almost always; 2 Yes, frequently; 3 Yes, sometimes; 4 Yes, seldom; 5 No, almost never. Higher scores mean a worse outcome. | 18 months | 
| Non-instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease. | Non-instrumental assessment and symptoms of dysphagia using the Bedside Swallowing Assessment Scale. The Bedside Swallowing Assessment Scale interpretation: Level 1: irrelevant alterations, scores ≤ 19; level 2: average alterations, scores 20-23; level 3: severe alterations, scores ≥ 24. Higher scores mean a worse outcome. | 18 months | 
| Instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease. | Assessment of pharyngeal and esophageal dysphagia using the Videofluoroscopic Swallowing Study, also known as the Modified Barium Swallow Study. The instrumental assessment provides a description of any anatomical differences and physiological problems that may be associated with the patients' symptoms in all phases of the swallow. Results from the videoflourographic study are used to rate the severity of dysphagia according to the Dysphagia Outcome and Severity Scale. The Dysphagia Outcome and Severity Scale scores range from 1 to 7, with 7 representing normal swallowing and 1 representing severe dysphagia. | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effect of deutetrabenazine on body weight in HD patients. | Secondary endpoints are treatment success from baseline to maintenance therapy on body weight. Body weight will be calculated in kilograms. | 18 months | 
| Effect of deutetrabenazine on body mass index in HD patients. | Secondary endpoints are treatment success from baseline to maintenance therapy on body mass index. Body mass index will be calculated by dividing the weight in kilograms by the square of the height in meters (kg/m2). | 18 months | 
| Effect of deutetrabenazine on fat distribution in HD patients. | Secondary endpoints are treatment success from baseline to maintenance therapy on body fat distribution. Body fat distribution will be estimated using skinfold caliper measurements of skinfold thickness at four sites: scapular, biceps, triceps and suprailiac, together with arm and leg circumferences. | 18 months |