Abstract Details

Title Valbenazine Decreases Disease Burden in Patients with Chorea Associated with Huntington Disease: Patient-Reported Huntington Disease Health Index (HD-HI) Results From the KINECT-HD Trial

Topic Movement Disorders

Presentation(s) P2 - Poster Session 2 (11:45 AM-12:45 PM)

Poster/Presentation
Number 5-012

Objective

To describe results of the Huntington Disease Health Index (HD-HI) in KINECT-HD (NCT04102579).

Background

In KINECT-HD, adults with Huntington disease (HD)-related chorea received placebo or valbenazine for 12 weeks. Top-line results demonstrated that once-daily valbenazine significantly improved chorea and was well tolerated (AAN 2022). KINECT-HD is the first Ph3 trial to implement the HD-HI, a novel, multidimensional, patient-reported outcome (PRO) validated to assess HD-related disease burden.

Design/Methods

HD-HI includes 13 subscales (score range: 0=no disease burden to 100=highest disease burden) and a total score. Mean HD-HI scores at baseline, Wk10, and Wk12 were analyzed descriptively. Exploratory ANCOVA analyses were performed post hoc for HD-HI score changes from baseline.

Results

Greater numerical decreases from baseline to Wk10 and Wk12 in HD-HI subscales were noted for valbenazine versus placebo in mobility (Wk10: -6.3 vs -4.1; Wk12: -4.2 vs -2.7), abnormal movements (Wk10: -12.2 vs -9.6; Wk12: -11.2 vs -4.3), and hand/arm function (Wk10: -7.2 vs -1.6; Wk12: -7.2 vs -1.4); however, baseline values were slightly higher in the valbenazine group for mobility and hand/arm function. Changes from baseline to Wk10 and Wk12 for fatigue and gastrointestinal health (including swallowing function) were unremarkable for valbenazine but also indicated no worsening. No meaningful changes in HD-HI total score were noted at Wk10 and Wk12. Least squares (LS) mean changes from baseline to Wk12 in the HD-HI abnormal movements subscale were -11.1 for valbenazine and -4.4 for placebo, with an LS mean difference between treatment groups of -6.7 (nominal P=0.0379).

Conclusions

HD-HI results from KINECT-HD demonstrated the successful implementation of an HD-specific PRO during serial evaluation in the context of a Ph3 trial, along with reduced disease burden with valbenazine for abnormal movements, mobility, and hand/arm function in patients with HD-related chorea. This highlights the importance of monitoring changes in patient-reported disease burden in response to therapeutic interventions.

Authors/Disclosures
Dietrich Haubenberger, MD, FAAN (Neurocrine Biosciences) PRESENTER	Dr. Haubenberger has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Haubenberger has stock in Neurocrine Biosciences. Dr. Haubenberger has a non-compensated relationship as a Member of the Board with American Society for Experimental Neurotherapeutics that is relevant to AAN interests or activities.
Chad R. Heatwole, MD, FAAN (University of Rochester Medical Center)	Dr. Heatwole has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Virginia Commonwealth University. Dr. Heatwole has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Legal Med. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurocrine. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Swan Bio. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Harmony. Dr. Heatwole has received personal compensation in the range of $0-$499 for serving as a Consultant for Iris. Dr. Heatwole has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Recursion. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity Biosciences. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lupin. Dr. Heatwole has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurocrine. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for New York Central Mutual. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Penn Prop and Gas. The institution of Dr. Heatwole has received research support from Department of Defense. The institution of Dr. Heatwole has received research support from Novartis. The institution of Dr. Heatwole has received research support from MJFF. The institution of Dr. Heatwole has received research support from FARA. The institution of Dr. Heatwole has received research support from NIH. The institution of Dr. Heatwole has received research support from University of Miami. The institution of Dr. Heatwole has received research support from MDA. Dr. Heatwole has received intellectual property interests from a discovery or technology relating to health care.
Erin Furr-Stimming, MD, FAAN (University of Texas Health Science Center-Houston)	Dr. Furr-Stimming has received personal compensation for serving as an employee of Help4HD International. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medscape. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MedPage. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Wave Life Sciences. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Latus Bio. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Atalanta Therapeutics. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SkyHawk Therapeutics. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis . Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Atalanta Therapeutics. The institution of Dr. Furr-Stimming has received research support from Roche/Genetech. The institution of Dr. Furr-Stimming has received research support from Uniqure. The institution of Dr. Furr-Stimming has received research support from CHDI. The institution of Dr. Furr-Stimming has received research support from Huntington Study Group/Neurocrine Bioscienes. The institution of Dr. Furr-Stimming has received research support from NIH/University of Iowa. The institution of Dr. Furr-Stimming has received research support from Sage Therapeutics. The institution of Dr. Furr-Stimming has received research support from HDSA. The institution of Dr. Furr-Stimming has received research support from Prilennia. Dr. Furr-Stimming has received publishing royalties from a publication relating to health care. Dr. Furr-Stimming has received publishing royalties from a publication relating to health care. Dr. Furr-Stimming has a non-compensated relationship as a Committee member with AAN UES Committee that is relevant to AAN interests or activities.
Daniel O. Claassen, MD, FAAN (Vanderbilt University Medical Center)	Dr. Claassen has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Alterity. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lundbeck. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Teva. Dr. Claassen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for AskBio. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for University of Michigan. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cognition Therapeutics . Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx. The institution of Dr. Claassen has received research support from NIH. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from HDSA. The institution of Dr. Claassen has received research support from Department of Defense. The institution of Dr. Claassen has received research support from CHDI.
Elise P. Kayson, RN	Elise Paulin Kayson, RN has nothing to disclose.
Jody Goldstein (Huntington Study Group)	No disclosure on file
Olga Klepitskaya, MD, FAAN (Neurocrine Biosciences, Inc)	Dr. Klepitskaya has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Klepitskaya has or had stock in Neurocrine Biosciences.
Grace L. Liang, MD	Dr. Liang has received personal compensation for serving as an employee of Neurocrine Biosciences. Dr. Liang has stock in Neurocrine Biosciences.

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