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Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated With HD

Primary Purpose

Huntington Disease

Status
Unknown status
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Deutetrabenazine Oral Tablet [Austedo]
Placebo oral tablet
Sponsored by
Fundacion Huntington Puerto Rico
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Huntington Disease

Eligibility Criteria

21 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    Placebo Comparator

    Arm Label

    Deutetrabenazine

    Placebo

    Arm Description

    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.

    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.

    Outcomes

    Primary Outcome Measures

    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.
    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.
    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.

    Secondary Outcome Measures

    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.
    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).
    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.

    Full Information

    First Posted
    February 13, 2020
    Last Updated
    July 20, 2020
    Sponsor
    Fundacion Huntington Puerto Rico
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04301726
    Brief Title
    Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated With HD
    Official Title
    Efficacy of Deutetrabenazine in Huntington's Disease Patients With Dysphagia: A Randomised, Placebo-controlled Pilot Study
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    July 2020
    Overall Recruitment Status
    Unknown status
    Study Start Date
    September 1, 2020 (Anticipated)
    Primary Completion Date
    April 30, 2022 (Anticipated)
    Study Completion Date
    December 31, 2022 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Fundacion Huntington Puerto Rico

    4. Oversight

    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
    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Huntington Disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Interventional Study Model
    Parallel Assignment
    Model Description
    Placebo-controlled pilot study
    Masking
    ParticipantInvestigatorOutcomes Assessor
    Allocation
    Randomized
    Enrollment
    48 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Deutetrabenazine
    Arm Type
    Experimental
    Arm Description
    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.
    Arm Title
    Placebo
    Arm Type
    Placebo Comparator
    Arm Description
    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.
    Intervention Type
    Drug
    Intervention Name(s)
    Deutetrabenazine Oral Tablet [Austedo]
    Other Intervention Name(s)
    Austedo
    Intervention Description
    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).
    Intervention Type
    Drug
    Intervention Name(s)
    Placebo oral tablet
    Other Intervention Name(s)
    Placebo
    Intervention Description
    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).
    Primary Outcome Measure Information:
    Title
    Efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease.
    Description
    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.
    Time Frame
    18 months
    Title
    Non-instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease.
    Description
    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.
    Time Frame
    18 months
    Title
    Instrumental assessment of the efficacy of deutetrabenazine to control symptoms of dysphagia associated with Huntington's disease.
    Description
    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.
    Time Frame
    18 months
    Secondary Outcome Measure Information:
    Title
    Effect of deutetrabenazine on body weight in HD patients.
    Description
    Secondary endpoints are treatment success from baseline to maintenance therapy on body weight. Body weight will be calculated in kilograms.
    Time Frame
    18 months
    Title
    Effect of deutetrabenazine on body mass index in HD patients.
    Description
    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).
    Time Frame
    18 months
    Title
    Effect of deutetrabenazine on fat distribution in HD patients.
    Description
    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.
    Time Frame
    18 months
    Other Pre-specified Outcome Measures:
    Title
    Effect of deutetrabenazine on quality of life in HD patients.
    Description
    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.
    Time Frame
    18 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    21 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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

    12. IPD Sharing Statement

    Learn more about this trial

    Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated With HD

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