Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated With HD

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

To determine the efficacy of deutetrabenazine to control symptoms of dysphagia associated with HD.

Study Overview

• Status: Not yet recruiting
• Conditions: Huntington Disease
• Intervention / Treatment: Drug: Deutetrabenazine Oral Tablet [Austedo]; Drug: Placebo oral tablet

Detailed Description

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

• Study Type: Interventional
• Enrollment (Anticipated): 48
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Deutetrabenazine; 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]; 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).; Other Names: Austedo
Placebo Comparator: Placebo; 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; 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).; Other Names: Placebo

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of deutetrabenazine on quality of life in HD patients.	Treatment success from baseline to maintenance therapy on quality of life of HD patients with dysphagia. The quality of life (specifically Assessment of health status and disability using the World Health Organization Disability Assessment Schedule (WHODAS) version 2.0. The WHODAS 2.0 covers six domains of functioning including cognition, mobility, self-care, getting along, life activities and participation in community activities. The following numbers are assigned to the responses: 0= No difficulty; 1= Mild difficulty; 2= Moderate difficulty; 3= Severe difficulty; and 4= Extreme difficulty or can't do. In this test of global day-to-day functioning, higher scores indicate worse functioning.	18 months

Collaborators and Investigators

• Sponsor: Fundacion Huntington Puerto Rico

Study record dates

Study Major Dates

• Study Start (Anticipated): September 1, 2020
• Primary Completion (Anticipated): April 30, 2022
• Study Completion (Anticipated): December 31, 2022

Study Registration Dates

• First Submitted: February 13, 2020
• First Submitted That Met QC Criteria: March 6, 2020
• First Posted (Actual): March 10, 2020

Study Record Updates

• Last Update Posted (Actual): July 21, 2020
• Last Update Submitted That Met QC Criteria: July 20, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Chorea; Huntington Disease; Physiological Effects of Drugs; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Neurotransmitter Uptake Inhibitors; Membrane Transport Modulators; Adrenergic Uptake Inhibitors; Tetrabenazine
• Other Study ID Numbers: 2002027824

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes