Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated with HD

NCT04301726 | Terminated Phase 1

• 1 other identifier: 2002027824
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Huntington Disease

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (3)

• Effect of deutetrabenazine on body weight in HD patients.

  18 months

• Effect of deutetrabenazine on body mass index in HD patients.

  18 months

• Effect of deutetrabenazine on fat distribution in HD patients.

  18 months

Other Outcomes (1)

• Effect of deutetrabenazine on quality of life in HD patients.

  18 months

Study Arms (2)

Deutetrabenazine

EXPERIMENTAL

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]

Placebo

PLACEBO COMPARATOR

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

Interventions

Deutetrabenazine Oral Tablet [Austedo] DRUG

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).

Also known as: Austedo

Deutetrabenazine

Placebo oral tablet DRUG

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).

Also known as: Placebo

Placebo

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of HD
• Symptoms of dysphagia
• Must be able to swallow tablets

You may not qualify if:

• Other confounding diseases that affect swallowing
• Depression
• Hepatic impairment
• Renal impairment
• Dementia

Sponsors & Collaborators

• Fundacion Huntington Puerto Rico: lead

Study Sites (1)

Fundacion Huntington Puerto Rico, Inc. - Huntington's disease Clinic

San Juan, 00926, Puerto Rico

Location

MeSH Terms

Conditions

Huntington Disease

Interventions

deutetrabenazine

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Scientific Official/Principal Investigator

Model Details: Placebo-controlled pilot study

Study Record Dates

• First Submitted: February 13, 2020
• First Posted: March 10, 2020
• Study Start: January 21, 2022
• Primary Completion: August 18, 2023
• Study Completion: September 1, 2023
• Last Updated: February 27, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

PR (1)