Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated with HD

Phase 1

Terminated

• Conditions: Huntington Disease
• Interventions: Drug: Placebo oral tablet; Drug: Deutetrabenazine Oral Tablet [Austedo]

• Registration Number: NCT04301726
• Lead Sponsor: Fundacion Huntington Puerto Rico

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.

Study Timeline

• Study First Submitted: 2020-02-13
• Study First Submitted QC: 2020-03-06
• Study First Posted: 2020-03-10
• Study Start: 2022-01-21
• Primary Completion: 2023-08-18
• Study Completion: 2023-09-01
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-25
• Last Update Posted: 2025-02-27

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 36

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo oral tablet	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.
Deutetrabenazine	Deutetrabenazine Oral Tablet [Austedo]	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.

Primary Outcome Measures

Name	Time	Method
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.
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.
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 Outcome Measures

Name	Time	Method
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).
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.

Trial Locations

Locations (1)

Fundacion Huntington Puerto Rico, Inc. - Huntington's disease Clinic; 🇵🇷 San Juan, Puerto Rico