Huntington's Disease Society of America (HDSA) Center of Excellence at the Neurological Institute of New York

Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease. Duration: 112 days. Participants must have a total functional capacity score (TFC) ranging from 7-12 (<6 and 13 excluded) and a MoCA score <26 at the time of enrollment. Assessments will include cognitive tests, physical examinations, psychiatric assessments, Virtual Reality Functional Capacity Assessment Tool (VRFCAT), and blood tests.

PI: Dr. O’Shea; Call Jasim Uddin 212-305-4597

Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Effect of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease. Duration: 84 days. Participants must have a total functional capacity score (TFC) ranging from 7-12 (<6 and 13 excluded) and a MoCA score <26 at the time of enrollment. Assessments will include cognitive tests, physical examinations, psychiatric assessments, Virtual Reality Functional Capacity Assessment Tool (VRFCAT), and blood tests.

PI: Dr. O’Shea; Call Jasim Uddin 212-305-4597

A Phase 3, multicenter, open-label safety study to evaluate the long-term safety and tolerability of SAGE-718 in participants with Huntington’s disease. We are recruiting individuals with HD who participated in SAGE 201 as well as new participants. Duration of the study will be one year.

PI: Dr. O’Shea. Call Jasim Uddin 212-305-4597

Movement Intervention for Neurodegenerative Disease (MIND): Dance for HD Phase 2. Feasibility and preliminary efficacy study. Inclusion: Diagnosed with HD. Age >18. TFC score= 7-13. Intervention= Laban-Bartenieff dance technique, 2 days a week for 18 weeks. Duration: about 24 weeks. Visits: 4 (pre-assessment, baseline, post-assessment, retention). Procedures include: Neurological assessment, balance and gait test, cognitive test, quality of life questionnaire.

PI: Dr. Rao. Call Corey Landis 212-305-1647

Enroll-HD is a longitudinal, observational, multinational study whose goal is to develop a comprehensive repository of prospective and systematically collected clinical research data (demography, clinical features, family history, genetic characteristics) and biological specimens (blood) from individuals with manifest HD, unaffected individuals known to carry the HD mutation or at risk of carrying the HD mutation, and control research participants (e.g., spouses, siblings or offspring of HD mutation carriers known not to carry the HD mutation). More than 20,000 participants have now enrolled into the study for annual assessments (www.enroll-hd.org/for-researchers/access-data/) to learn more.

PI: Karen Marder. Call Jasim Uddin 212 305 -4597

Child to Adult Neurodevelopment in Gene Expanded Huntington’s Disease

Duration: 5 years, yearly visit Inclusion: Ages 6-30 with parent/grandparent with HD, Procedures: blood draw, structural MRI, lifestyle questionnaires, cognitive tests, motor tests.

PI: Dr. Rao. Call Jennifer Oscherician 212-305-1647

Individuals with Huntington's disease (HD) have balance impairments that put them at risk for falls, however there are no standardized outcome measures of balance that are specific to HD. Outcome measures are vital to understanding the nature of balance impairments as well as to evaluate improvement in balance following interventions, including medication or rehabilitation. The purpose of this study is to administer a battery of balance outcome measures that have been used in people with neurodegenerative diseases to determine which items are appropriate for people with HD. This study requires 1 in person assessment (with a second optional in person assessment one week later). The study time takes 2 hours each session, and participants will undergo some patient-reported assessments as well as testing of balance and walking. This study will take place at Teachers College, Columbia University at 525 W 120th St. CUMC IRB: AAAU1786.

For information, contact Chelsea Macpherson, PT, DPT, NCS at 212-678-3916 or neurorehablab@tc.columbia.edu