Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease

Phase 3

Completed

• Conditions: Chorea, Huntington
• Interventions: Drug: Valbenazine; Drug: Placebo

• Registration Number: NCT04102579
• Lead Sponsor: Neurocrine Biosciences

Brief Summary

This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of valbenazine to treat chorea in participants with Huntington disease.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-09-23
• Study First Submitted QC: 2019-09-23
• Study First Posted: 2019-09-25
• Study Start: 2019-11-13
• Primary Completion: 2021-10-15
• Study Completion: 2021-10-26
• Disposition First Submitted: 2022-09-29
• Disposition First Submitted QC: 2022-09-29
• Disposition First Posted: 2022-10-05
• Status Verified: 2023-09
• Results First Submitted: 2023-09-15
• Results First Submitted QC: 2023-09-15
• Last Update Submitted: 2023-09-15
• Results First Posted: 2023-10-11
• Last Update Posted: 2023-10-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 128

Inclusion Criteria

1. Have a clinical diagnosis of Huntington Disease (HD) with chorea
2. Be able to walk, with or without the assistance of a person or device
3. Participants of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently while participating in the study until 30 days (females) or 90 days (males) after the last dose of the study drug
4. Be able to read and understand English

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Exclusion Criteria

1. Have a history of previously established therapy with a VMAT2 inhibitor, in the judgement of the investigator
2. Have difficulty swallowing
3. Are currently pregnant or breastfeeding
4. Have a known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular block, uncontrolled bradyarrhythmia, or heart failure
5. Have an unstable or serious medical or psychiatric illness
6. Have a significant risk of suicidal behavior
7. Have a history of substance dependence or substance (drug) or alcohol abuse, within 1 year of screening
8. If taking antidepressant therapy, be on a stable regimen
9. Have received gene therapy at any time
10. Have received an investigational drug in a clinical study within 30 days of the baseline visit or plan to use such investigational drug (other than valbenazine) during the study
11. Have had a blood loss ≥550 milliliters (mL) or donated blood within 30 days before the baseline visit
12. Had a medically significant illness within 30 days before baseline, or any history of neuroleptic malignant syndrome

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Valbenazine	Valbenazine	Capsule, administered orally once daily for 12 weeks.
Placebo	Placebo	Capsule, administered orally once daily for 12 weeks.

Primary Outcome Measures

Name	Time	Method
Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.	Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)	The TMC is part of the motor assessment of the UHDRS and measures chorea in 7 different body parts including the face, oral-buccal-lingual region, trunk and each limb independently. The TMC score is the sum of the individual scores and ranges from 0 to 28. A decrease in TMC scores indicates improvement in chorea symptoms.

Secondary Outcome Measures

Name	Time	Method
Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12	Week 12	The CGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the investigator or qualified clinician designee.; Participants whose CGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12	Week 12	The PGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the participant.; Participants whose PGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score	Baseline, Week 12	The Neuro-QoL Upper Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates increased function.
Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score	Baseline, Week 12	The Neuro-QoL Lower Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates better function.

Trial Locations

Locations (1)

Neurocrine Clinical Site; 🇨🇦 Toronto, Ontario, Canada