A Clinical Study for Huntington’s Disease Chorea

ABOUT HUNTINGTON DISEASE

One of the defining symptoms of HD is chorea, which is characterized by abnormal, abrupt, irregular movements. As HD progresses, chorea can become more frequent and severe, negatively impacting quality of life. While there is no established treatment to delay onset or progression of HD, a product called valbenazine is being evaluated as a potential treatment for HD-associated chorea.

PURPOSE OF THE KINECT-HD2 STUDY

Valbenazine (valbenazine tosylate, NBI-98854) is a selective, orally active VMAT2 inhibitor developed by Neurocrine Biosciences, Inc. that is being investigated for the treatment of chorea associated with Huntington’s disease (HD). The study drug, valbenazine, has been approved by the United States Food and Drug Administration (FDA) in April 2017 for the treatment of adults with tardive dyskinesia (TD), under the trade name INGREZZA®.

KINECT-HD2 is an Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated with Huntington’s Disease.

All subjects in Study NBI-98854-HD3006 will self-administer a once-daily (qd) dose of valbenazine 20 mg to 80 mg for up to 104 weeks.

The objectives of KINECT-HD2 are to:

• Evaluate the long-term safety and tolerability of valbenazine, administered once daily (qd) for up to 104 weeks in subjects with Huntington’s disease
• Evaluate long-term maintenance of effect of valbenazine for the treatment of chorea associated with HD in approximately 150 subjects with HD.

WHO MAY QUALIFY?

This study will enroll approximately 150 medically stable male and female subjects with genetically confirmed motor manifest HD; approximately 120 of which will rollover from the KINECT-HD study (NBI-98854-HD3005). Subjects who early terminated Study NBI-98854-HD3005 for administrative reasons due to coronavirus disease 2019 (COVID-19) may also enroll into the KINECT-HD2 study.

Eligible participants must meet the following criteria, in addition to other criteria:

• Male or female, ages of 18 to 75 years
• Diagnosis of motor manifest HD
• Have sufficient chorea symptoms to meet study protocol criteria
• Willing and able to comply with the study instructions

There are additional eligibility requirements that the Clinical Investigator can explain to you.

STUDY SCHEDULE

Participation in the KINECT-HD2 study will last up to 112 weeks, but study participants are not required to stay in the study for its entire duration. The intent of the duration is to provide access to valbenazine until it is anticipated to be available commercially for the treatment of chorea associated with HD, subject to approval by the FDA and any other applicable regulatory authorities. The study includes a screening period, a study drug period, and a follow-up period. Participants will attend eighteen (18) in-person study visits throughout the study. An additional five (5) visits will be conducted by phone or video call.

Subjects rolling over from KINECT-HD (NBI-98854-HD3005)

Subjects rolling over from KINECT-HD (NBI-98854-HD3005) have some additional options for timing initial KINECT-HD2 visits with those of KINECT-HD. Subjects rolling over into KINECT-HD2 should speak with their Study Site Investigator or Study Site Coordinator as soon as possible to discuss and understand all available scheduling options.

Subjects who did not participate in KINECT-HD (NBI-98854-HD3005)

Subjects who did not participate in the KINECT-HD Study (NBI-98854-HD3005) must complete all screening assessments and assessments required at baseline.

Screening

The screening period is only applicable for subjects who did not rollover from KINECT-HD (NBI-98854-HD3005) or those who had their final KINECT-HD visit >30 days prior to anticipated baseline (Day -1) for KINECT-HD2 (NBI-98854-HD3006), or had been early terminated for administrative reasons due to COVID-19 from KINECT-HD.

After you consent to participate in the study, you will need to attend a screening visit, where your Clinical Investigator will perform tests and procedures to confirm your eligibility to take part in the study. The screening portion of the study will last up to four (4) weeks.

Study Drug Dosing

If you qualify based on the study eligibility criteria, you will be in this part of the study for up to 104 weeks. During this part of the study, you will first enter the dose adjustment period for eight (8) weeks. During the dose-adjustment period, the Clinical Investigator will increase your dose to the next dose level if, in the Clinical Investigator’s opinion, you have tolerated the study drug at the current dose.

Following the 8-week dose adjustment period, you will enter a dose maintenance period until Week 104. During the maintenance period, your dose of the study drug valbenazine will be maintained. However, if you are not able to tolerate the study drug, Clinical Investigator may adjust the dose to be lower.

Follow-Up

A final checkup will be done at Week 108.

HOW TO PARTICIPATE IN THIS CLINICAL TRIAL

KINECT-HD2 is being conducted by the Huntington Study Group (HSG), a leader in HD clinical trials for over 25 years. KINECT-HD2 will take place in approximately 45 study centers in the United States and Canada.

If you are interested in participating in KINECT-HD2, please contact the site nearest to you. Study center personnel will determine your eligibility to participate in the KINECT-HD2 study. You can also contact the Huntington Study Group to inquire about KINECT-HD2.

Call Toll-Free (North America): 800-487-7671
Email: info@hsglimited.org

Frequently Asked Questions (FAQs)

Valbenazine (valbenazine tosylate, NBI-98854) is a selective, orally active VMAT2 inhibitor developed by Neurocrine Biosciences, Inc. Valbenazine is being investigated for the treatment of chorea associated with HD. Valbenazine was approved by the United States Food and Drug Administration (FDA) in April 2017 for the treatment of adults with tardive dyskinesia (TD), under the trade name INGREZZA®.

Twenty-seven clinical studies with valbenazine have been completed to date: 16 Phase 1 studies in healthy subjects or special populations (for example, patients with impaired liver or kidney function); 7 Phase 2 or 3 studies in subjects with TD, and 4 Phase 1b or 2 studies in subjects with Tourette syndrome (TS; 1 in adults; 2 in children and adolescents; and 1 in children, adolescents, and adults).

Most commonly-reported side effects with valbenazine administration include drowsiness, tiredness and sedation (feeling calm, relaxed or sleepy).

Valbenazine is being evaluated as a symptomatic treatment of involuntary movement (chorea) associated with HD. Valbenazine is not an approved treatment or cure for HD.

Valbenazine was approved by the United States Food and Drug Administration (FDA) in April 2017 for the treatment of adults with tardive dyskinesia (TD), under the trade name INGREZZA®.

Yes. KINECT-HD2 is actively seeking to enroll 30 additional participants that were not part of the KINECT-HD double-blind study. Approximately 120 KINECT-HD (NBI-98854-HD3005) participants are anticipated to rollover, for a total of approximately 150 total subjects in KINECT-HD2.

Yes. There are several requirements that a participant must meet to be enrolled in KINECT-HD2, including the following:

• Be male or female, ages of 18 to 75 years
• Have a diagnosis of motor manifest HD
• Have sufficient chorea symptoms to meet study protocol criteria
• Is willing and able to comply with the study instructions

There are additional eligibility requirements that the Clinical Investigator can explain to you.

Participants enrolled in KINECT-HD2 will participate up to 112 weeks. The participation timeline includes a 4-week screening period for subjects who did not participate in KINECT-HD, an 8-week dose-adjustment period, a 96-week maintenance period, and a follow-up visit two weeks later.

KINECT-HD2 is an open-label rollover study. This means that participants will be dosed on study drug. There is no placebo in the open-label study (OLE). Valbenazine will be supplied as orally administered capsules containing 20, 40, 60, or 80 mg of valbenazine. Subjects must swallow the capsules with approximately 4 ounces of water or other liquid, with or without food.

Yes. All participants must provide their written informed consent before being screened and participating in the study.

The KINECT-HD2 study is being conducted by the Huntington Study Group on behalf of Neurocrine Biosciences, Inc. Interested participants are encouraged to speak with their doctor about the study and may contact the Huntington Study Group to find the nearest participating site.

Toll-free phone (North America): 800-487-7671
Email: info@hsglimited.org

What You Should Know About Clinical Research Studies

WHAT IS A CLINICAL RESEARCH STUDY?

Clinical research studies, also called clinical trials, are research studies in which participants are asked to take a study drug under the supervision of a physician and other research professionals.

Clinical research studies must be approved by an institutional review board (IRB). An IRB is a group that is responsible for helping to protect the rights and welfare of study participants. In addition, every study participant is monitored with study-required medical tests and exams before, during, and sometimes even after the study.

CAN I LEAVE THE STUDY IF I CHANGE MY MIND?

Participation in any clinical research study is completely voluntary, and participants may choose to leave the study at any time for any reason. If you would like to leave the study, you should discuss this with your Clinical Investigator, who will give you information about how to do this safely.

KINECT-HD2 STUDY LOCATIONS

KINECT-HD2 will be conducted at several of the same research sites which conducted KINECT-HD.

If you are interested in participating in KINECT-HD2, you may contact the Huntington Study Group to find the nearest available participating KINECT-HD2 research site.