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Current Research – Observational Studies

Multicenter Observational Studies (Huntington Study Group)

COHORT: Cooperative Huntington’s Observational Research Trial

The COHORT study is open to individuals who are affected by HD and those who are part of an HD family. The purpose of this study is to collect data over time to uncover information about the natural history and add to the scientific knowledge about HD.  Ultimately, the goal of this ambitious project is to aid in the development of potential treatments for HD.  We began enrolling participants in COHORT in May, 2006.  If you would like more information or would like to participate, please contact Ronda Clouse, RN at (212) 305-2387 or email

PREDICT: Neurobiological Predictors of Huntington's Disease

PREDICT was developed to follow individuals who know they carry the HD gene in order to determine the earliest signs of HD, identify the most sensitive tests for clinicians to detect these symptoms, and what factors influence the age at which a person carrying the HD gene develops symptoms.   Clinical evaluations are done annually.  For more information about PREDICT, please check the Huntington Study Group website. This study is closed to new enrollment at Columbia University. 


PHAROS was developed to follow individuals who know that they are at risk for developing HD but choose not to know their genetic status.  We first enrolled subjects in 1999, and have interviewed and examined participants every 9 months.  We continue to follow 30 individuals and hope to extend this study through to the year 2010.  For more information about PHAROS, please check the Huntington Study Group website. This study is closed to new enrollment.


This observational study is linked to the PREDICT and PHAROS studies.   This study will examine the experiences of individuals who have undergone genetic testing for HD or who have a family history of HD.  A survey will be administered to assess stigmatization and discrimination individuals may have experienced, and question how individuals perceive possible consequences, choose their behaviors and make life decisions after genetic testing or because of a family history of HD.   Enrollment is currently underway.  If you would like more information, please contact Paula Leber Wasserman at (212) 305-4597 or

Investigator-Initiated Observational Trials at Columbia University

PHAIMOS: Prospective HD mutation Assessment using Indices of Motor Skill

In this study we plan to identify early motor manifestations of the HD gene mutation by testing performance on motor learning tasks. The goal is to find performance differences between different groups by combining motor learning and working memory requirements in a single task. Subjects move a cursor on a computer monitor using a computer mouse and learn to adapt to changes in cursor movement imposed by the computer. Subjects include a group of asymptomatic gene-positive individuals, individuals with mild symptoms of HD, and a group of control subjects. Please contact Pietro Mazzoni, MD, PhD at (212) 305-0737. This study is funded through an Investigator Grant to Dr. Mazzoni from the Huntington's Disease Society of America.

Current Research – Clinical Trials

A clinical trial, or drug study, is the most effective method for estimating the medical value of promising treatments. By design, it provides the necessary confidence and safeguards for learning whether medications do what they are supposed to do. Each clinical trial tests a compound that has shown promise in HD transgenic mouse-models, in small pilot clinical studies and in lower doses in earlier clinical studies with people who have HD.

A compound can be any potentially effective therapeutic agent: vitamins, health food supplements, or medications. There are different types of clinical trials to answer different questions: What is the ideal daily dose? Is the ideal daily dose safe and easily tolerated? Is the ideal daily dose effective in slowing down progression of HD?

When a new compound shows promise, the compound needs to be tested in people with HD to find the ideal dose per day. These are called dose-finding studies and usually recruit approximately 30 participants at one medical center. Then the compound needs to be tested for safety and tolerability at specific doses. These clinical trials are called safety and tolerability studies, usually recruit approximately 64 participants at approximately 4 medical centers. Then, having passed these tests, the compound is tested in an efficacy study that usually recruits hundreds of volunteer participants in many medical centers over many months.

Clinical Trials at Columbia University


2CARE is a 5 year long study enrolling participants with early HD across the U.S. and Canada. The purpose is to understand whether or not high dose of Co-enzyme Q-10 can slow down the worsening of HD. 2CARE is a follow-up study to the CARE-HD study in which Co-enzyme Q-10 at 600 mg daily was not effective enough to be statistically significant.  In 2CARE, Co-Q 10 will be provided in a higher dose for a longer duration because there was a trend toward significance in the initial CARE-HD study. Enrollment will begin in Early 2008.


DIMOND is a 104 day study for participants with early HD who live in the USA or UK.   The purpose is to measure the safety and tolerability of Dimebon, a medication that has been used to treat allergies, when taken for 90 days.  This is an industry sponsored study of a medication that may improve cognitive function.  Enrollment will begin in late Fall 2007.


DOMINO is an 18-month trial to test the need to launch a larger and longer study of minocycline, an FDA approved antibiotic, whose other action is to slow down the process of programmed cell death, called apoptosis.  Study subjects will take the active drug or placebo for 18 months.  Enrollment began in Summer 2006. This study is closed to enrollment. 

Interested in participating?

  • People who are currently patients at the Center may call Carol Moskowitz (212) 305-5779 or email to get an update and ask to be informed when a study is about to start. 
  • Individuals that are not patients at the Center but would like to be considered for enrollment in clinical trials, please call Paula Leber Wasserman (212) 305-4597 for a new patient appointment.
  • Anyone from an HD family and would like to hear more about participation in observational studies is invited to call Paula Leber Wasserman at (212) 305-4597 or email  

Recently Completed - Observational Research


An observational study on energy and metabolism in HD, performed at Columbia University's HD Center of Excellence and St. Luke's Hospital, has been completed. The purpose of the study was to learn more about calorie needs of HD patients and how to maintain a healthy weight.  Results from this research can be seen in an Article published in the HDSA's Marker (pdf article link)  Please see Publications (see publication) for further information.

Functional Imaging in HD

The goal of the fMRI study conducted by Drs. Scott Small and Yvette Bordelon is to use a novel functional MRI technique to determine whether there are differences in distinct brain regions and brain metabolism in symptomatic and pre-symptomatic subjects when compared to healthy controls. Preliminary analysis of the data revealed that cerebral brain volume (CBV) measurement using this MRI technique may be a more sensitive marker of early neuronal dysfunction in HD.

Views of Privacy of Genetic and Other Health Information

The purpose of this study conducted by Dr. Robert Klitzman was to learn about the experiences of individuals who have or are at risk for genetically related disorders--including Huntington's Disease--concerning their views of privacy of medical information and attitudes about tests for genetic diseases.  Participants were interviewed for approximately two hours at Columbia University.  The data from this study has been analyzed and findings have been reported in three journal articles thus far.  Please see Publications for further information on these articles.

Recently Completed – HSG Clinical Trials


PHEND-HD was a six-month trial sponsored by NIH to test the safety and tolerability of a drug, phenylbutyrate, in people with early HD. This drug has the potential to "turn off" the HD gene that synthesizes the abnormal protein.  In HD mice, disease progression was slowed 22%.  If safe and tolerable, an efficacy study will be considered to test the role of phenylbutyrate in neuroprotection for HD.  Data analysis over the summer should provide results in late Fall 2007.  This study is closed. 


TREND-HD was a year long trial (6 months double-blind, then 6 months open-label) designed to test the efficacy of a drug, ethyl-EPA, in reducing the severity of choreic movements in people with HD.  Prior clinical studies suggested that ethyl-EPA reduced the amplitude of chorea in people with HD.  This 43-center trial was funded by Amarin Neuroscience, Ltd in collaboration with the Huntington Study Group (HSG).  Preliminary analysis of the 6–month data indicated that ethyl-EPA had no effect on chorea. Analysis of the 12 month data is pending. Please see press-release for further information about this drug trial.


RID-HD was a 9 week drug study evaluating the safety, tolerability and effects of Riluzole in people who have early signs of Huntington's Disease. This study is closed.  Please see Publications for further information.


CARE-HD attempted to determine if the progression of HD can be slowed by treatment with Remacemide and/or Co-enyzme Q-10 This study is closed and forms the basis of the upcoming 2CARE study.  Please see Publications (#5 and #8) for further information.

For more information about current clinical trials, check out the Huntington Study Group website.

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Columbia University Medical Center : Department of Neurology : Last updated 27-Jun-2009