Huntington Disease
Conditions
Brief summary
To determine the efficacy of deutetrabenazine to control symptoms of dysphagia associated with HD.
Detailed description
The primary endpoint is the change in swallow function/dysphagia from baseline to maintenance therapy.
Interventions
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).
DRUGPlacebo 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).
Sponsors
Fundacion Huntington Puerto Rico
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Intervention model description
Placebo-controlled pilot study
Eligibility
Sex/Gender
ALL
Age
21 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease. | 18 months | 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. |
| Efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease. | 18 months | 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. |
| Non-instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease. | 18 months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Effect of deutetrabenazine on fat distribution in HD patients. | 18 months | 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. |
| Effect of deutetrabenazine on body weight in HD patients. | 18 months | Secondary endpoints are treatment success from baseline to maintenance therapy on body weight. Body weight will be calculated in kilograms. |
| Effect of deutetrabenazine on body mass index in HD patients. | 18 months | 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). |
Other
| Measure | Time frame | Description |
|---|---|---|
| Effect of deutetrabenazine on quality of life in HD patients. | 18 months | 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. |
Countries
Puerto Rico
Outcome results
None listed