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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

  • PROOF-HD: Phase 3 clinical trial investigating the drug pridopidine
  • uniQure: Phase 1/2 clinical trial investigating the drug AMT-130
  • HDClarity: A Multi-Site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease
  • Enroll-HD: A Prospective Registry Study in a Global Huntington Disease (HD) Cohort

PROOF-HD

PROOF-HD is a Phase 3 clinical trial investigating the drug pridopidine as a treatment for Huntington’s disease. This study will enroll up to 480 people internationally with early-stage HD. To enroll, participants must be 25 or older and have a confirmed diagnosis of early stage, adult-onset HD. During the trial, participants will be randomly selected to receive either an oral capsule of 45 mg pridopidine or a placebo, used for comparison, taken two times a day. The study will last between 65 to 78 weeks (15 to 18 months), with an optional open-label extension phase – in which all participants will be given pridopidine - for patients who would like to continue treatment thereafter. Pridopidine was previously studied in other clinical trials (PRIDE-HD and OPEN-HART), which showed that it was overall well-tolerated by patients. To learn more about PROOF-HD at our center, please contact Kia Ultz at kcarte23@jhmi.edu or 410-955-1349.

uniQure

uniQure is conducting a double-blinded Phase I/II gene-based therapy known as AMT-130. AMT-130 is administered directly into the brain tissue using a minimally invasive brain surgery technique. The main purpose of this study is to find a safe dose of AMT-130 in adults who have early stage HD. It will also look at how the body processes AMT-130 and how it might affect the progression of HD. This study is open to patients with a clinical diagnosis of early-manifest HD and between the ages of 25 to 65 years old. A total of 26 patients from the United States will be enrolled, with 16 receiving the treatment of AMT-130 under general anesthesia (“Treated Group”) and the remaining 10 not receiving the treatment (“Imitation Group”). The main part of the study lasts for 18 months, with additional annual visits out to 5 years for continued safety follow up. Procedures will include clinic visits, assessments of physical and neurological health, a neurosurgical procedure, lumbar punctures, brain scans, and samples from body fluids. More information from the uniQure website: http://www.uniqure.com/patients/phase-1-2-clinical-trial-of-amt-130.php. If interested in participating or to find out more about this study, please contact Mollie Jenckes at mjenckes@jhmi.edu or 410-294-6503.


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.

For information about this study, please contact: Kia Ultz (kcarte23@jhmi.edu)


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. 

For information about this study, please contact: Etta Bernard (formerly Goldman) (egoldm17@jhmi.edu)

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