Clinical Studies

Clinical studies can include experimental treatments, such as drug trials, and observational studies. Studies are often conducted under the auspices of the Huntington Study Group or may be sponsored by pharmaceutical and biotechnology companies or the National Institute of Health. The Center also conducts a number of trials on its own.

Currently Enrolling Studies

HDClarity: A Multi-Site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease

The main purpose of this observational study is to collect cerebrospinal fluid (CSF), the fluid that surrounds the brain and spinal cord, which is needed to study biomarkers to see which biomarkers influence HD’s pathophysiology and progression, as well as how biomarkers differ between HD and non-HD participants. CSF can be used to provide information about the brain and the nervous system that is impossible to obtain in any other way. CSF is collected by a procedure called a lumbar puncture or spinal tap. This is a commonly performed procedure that takes around 30 minutes. Family members with a positive or negative HD status, or family not related through blood (i.e. spouses and partners) may participate. In addition, biomarkers may help design and guide future research studies and clinical trials as well as help us better understand who will most likely benefit from a particular treatment.

Enroll-HD: A Prospective Registry Study in a Global Huntington Disease (HD) Cohort

Enroll-HD is a multi-site clinical observational study that assess its participants on an annual basis. There are over 13,000 active participants worldwide, at 148 sites in 15 countries. Types of assessments used includes cognitive, behavioral, motor, and physiological, as well as demographic information and HD family history. You will also have a blood draw. Both people with and without HD can participate – this includes pre-symptomatic and symptomatic patients, those with unknown HD status, and family members. Furthermore, this study leads as a platform for future HD clinical research. 

Current Studies

Signal: A Phase 2, multi-center, randomized, double-blind, placebo controlled study in subjects with late prodromal and early manifest Huntington’s disease (HD) to assess the safety, tolerability, pharmacokinetics, and efficacy of VX15/2503

The primary objective of Phase 2 is to evaluate the safety, tolerability and efficacy of monthly IV administration of VX15/2503 over 18 months and up to 36 months (subset of subjects) in Cohort B in subjects with late prodromal or early manifest HD. Efficacy assessments for Cohort B will include neuropsychological, clinical, motor, global measurement of change and imaging endpoints in a population of late prodromal and early manifest HD patients over 18 months and up to 36 months.  This trial is being conducted at approximately 30 sites across the US and Canada, and seeks to enroll 240 individuals.

For research information, please contact:

Jacqueline V. Bran, M.A., M.H.S.
Research Program Coordinator
410-614-9483 desk
410-955-8233 fax
jbran1@jhmi.edu