Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated With HD

Huntington Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04301726
• Other study ID #: 2002027824
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: September 1, 2020
• Est. completion date: December 31, 2022

Study information

• Verified date: July 2020
• Source: Fundacion Huntington Puerto Rico
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 48
• Est. completion date: December 31, 2022
• Est. primary completion date: April 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 21 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Huntington Disease

Intervention

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).
• 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).

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Fundacion Huntington Puerto Rico

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Primary	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
Primary	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
Primary	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	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
Secondary	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
Secondary	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