Clinical trial NCT04400331: Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
Official title: Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Chorea, Huntington
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
Collaborators: name: Huntington Study Group
First posted: 2020-05-22
Enrollment: 154
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine, and to provide participants continued access to valbenazine for the treatment of chorea associated with Huntington disease.

Detailed description: After completion of Week 156/early termination visit, participants in the US will be given the option to continue into an extended maintenance period for up to 104 weeks and participants in Canada will have the option to participate in a separate open-label study (Study NBI-98854-HD3022).

Eligibility criteria: Inclusion Criteria:

Either #1 or #2 must be met for inclusion eligibility.

1. Have participated in Study NBI-98854-HD3005 and

   a. Study drug dosing completion of Study NBI-98854-HD3005, as demonstrated by completed study drug dosing through the follow-up visit or early terminated Study NBI-98854-HD3005 for administrative reasons due to COVID-19 (for example, site closure related to COVID-19)
2. Did not participate in Study NBI-98854-HD3005 and

   1. Have a clinical and genetic diagnosis of Huntington Disease (HD) with chorea
   2. Be able to walk, with or without the assistance of a person or device
3. Be able to read and understand English and capable of providing consent to study participation or have a legally authorized representative providing consent and the participant providing assent
4. Participants of childbearing potential must agree to use contraception consistently while participating in the study until 30 days after last dose of the study treatment

Exclusion Criteria:

1. Have difficulty swallowing
2. Are currently pregnant or breastfeeding
3. Have a known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular (AV) block, uncontrolled bradyarrhythmia, or heart failure
4. Have an unstable or serious medical or psychiatric illness
5. Have a significant risk of suicidal behavior
6. Have a history of substance dependence or substance (drug) or alcohol abuse, within 1 year of screening
7. Have received gene therapy at any time
8. Have received an investigational drug in a clinical study (other than valbenazine) within 30 days before the baseline visit or plan to use such investigational drug (other than valbenazine) during the study
9. Have had a blood loss ≥550 mL or donated blood within 30 days before the baseline visit
10. Have a history of severe hepatic impairment or history of protocol specified hematologic abnormalities during the course of the NBI-98854-HD3005 study
11. Had a medically significant illness within 30 days before baseline, or any history of neuroleptic malignant syndrome
12. Have a known hypersensitivity to any component of the formulation of valbenazine
13. For participants who did not participate in NBI-98854-HD3005: have a history of VMAT2 inhibitor use within 30 days of baseline

Primary outcomes:
measure: Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
timeFrame: Up to 262 weeks
Secondary outcomes:
measure: Change from Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score
timeFrame: Up to 262 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Neurocrine Clinical Site
state: Alabama
city: Little Rock
country: United States
facility: Neurocrine Clinical Site
state: Arkansas
city: La Jolla
country: United States
facility: Neurocrine Clinical Site
state: California
city: Aurora
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Washington
country: United States
facility: Neurocrine Clinical Site
state: District of Columbia
city: Boca Raton
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Gainesville
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Atlanta
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Indianapolis
country: United States
facility: Neurocrine Clinical Site
state: Indiana
city: Iowa City
country: United States
facility: Neurocrine Clinical Site
state: Iowa
city: Wichita
country: United States
facility: Neurocrine Clinical Site
state: Kansas
city: Louisville
country: United States
facility: Neurocrine Clinical Site
state: Kentucky
city: New Orleans
country: United States
facility: Neurocrine Clinical Site
state: Louisiana
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Charlestown
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Omaha
country: United States
facility: Neurocrine Clinical Site
state: Nebraska
city: Rochester
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Williamsville
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Durham
country: United States
facility: Neurocrine Clinical Site
state: North Carolina
city: Columbus
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Toledo
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Pittsburgh
country: United States
facility: Neurocrine Clinical Site
state: Pennsylvania
city: Charleston
country: United States
facility: Neurocrine Clinical Site
state: South Carolina
city: Columbia
country: United States
facility: Neurocrine Clinical Site
state: South Carolina
city: Greenville
country: United States
facility: Neurocrine Clinical Site
state: South Carolina
city: Nashville
country: United States
facility: Neurocrine Clinical Site
state: Tennessee
city: Houston
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Burlington
country: United States
facility: Neurocrine Clinical Site
state: Vermont
city: Vancouver
country: Canada
facility: Neurocrine Clinical Site
state: British Columbia
city: Ottawa
country: Canada
facility: Neurocrine Clinical Site
state: Ontario
city: Toronto
country: Canada
facility: Neurocrine Clinical Site
state: Ontario
Study officials:
affiliation: Chief Medical Officer
name: Chief Medical Officer
role: STUDY_DIRECTOR
References: