THERAPEUTIC TRIALS
Updated: March 20, 2008
Please check back often. New sites will be added to this list as they secure the approval required to enroll study participants.The Huntington Study Group (HSG), under the direction of Merit Cudkowicz MD, MSc (Massachusetts General Hospital), Michael McDermott PhD and Karl Kieburtz MD MPH (University of Rochester), is conducting a multi-center, randomized, double-blind, placebo-controlled study of coenzyme Q10 (CoQ) in individuals with Huntington’s disease (HD) to assess the effects of CoQ on the progression of functional decline in HD, as well as the long-term safety and tolerability at the dosage studied. Coenzyme Q10, a naturally occurring substance in the body, is available for purchase over the counter as a nutritional supplement. CoQ is being studied as an investigational drug at a higher dosage than is currently available for purchase.
CoQ has been used to treat a variety of human disorders, including those involving the heart and circulatory system, cancer, muscular dystrophy, a muscle coordination disorder called ataxia, and other disorders. The most marked results seem to have occurred in patients with a preexisting inherited deficiency of CoQ, although the studies reporting this information are limited by their not being controlled clinical trials. However, no serious safety issues have been reported. Recent preliminary studies of CoQ in neurologic disorders such as Parkinson’s disease, Amyotrophic Lateral Sclerosis, and Huntington’s disease confirm the safety and tolerability of CoQ in daily dosages up to, including, and exceeding the dosage planned for the 2CARE study when used for a short time.
The 2CARE study will be the largest therapeutic clinical trial to date in Huntington’s disease. Six hundred eight research participants will be enrolled at approximately forty-six clinical sites in the US, Canada, and Australia.
DIMOND: A Multi-Center, Phase 2, Randomized, Double-Blinded, Placebo-Controlled Study of Dimebon in Subjects with Huntington’s Disease
The Huntington Study Group (HSG) is conducting a study of the research medication Dimebon in persons 29 years of age or older who have mild to moderate Huntington’s disease (HD). DIMOND is designed to determine safe and tolerable doses of Dimebon and also to determine the effect of Dimebon on cognitive (thinking) and motor (movement) signs and overall functioning of subjects with HD. Approximately 15 research centers in the United States and in the United Kingdom enrolled about 90 research subjects. The entire time on study drug will last about 3 months. The study enrolled research subjects with early to moderate signs of HD who are independently ambulatory (walking) and self-sufficient in activities of daily living, such as eating, dressing, and bathing. Enrollment began in the Summer of 2007, and was completed in March 2008. The study is sponsored by Medivation, Inc.
Please see the DIMOND Participating Site List.
If you are interested in learning more about this study,
please contact the Huntington Study Group toll free at 1-800-487-7671,
or see the
July 2007 DIMOND press release or the
October 2006 DIMOND press release.Please also see the January 2008 article written by Marsha Miller, PhD, which is on the HDSA Web Site.
DOMINO [A Multi-Center, Double-Blind, Pilot Study of Minocycline in Huntington’s Disease]
The Huntington Study Group (HSG), under the direction of Merit Cudkowicz MD, MSc (Massachusetts General Hospital) and Karl Kieburtz MD, MPH (University of Rochester), is conducting a multi-center, randomized, double-blind study of minocycline in individuals with Huntington’s disease (HD) to assess long-term safety and to gather more information on its possible efficacy. Minocycline is a drug that has been used for the past 30 years to treat a variety of infections. Minocycline also has anti-cell death and anti-oxidant properties and may have potential therapeutic benefit in HD. Recent studies in a mouse model of HD have demonstrated that minocycline helps to slow down the clinical onset of HD and prolongs life. In a previous HSG trial led by Drs. Cudkowicz, Frederick Marshall and Robert Friedlander, minocycline was shown to be safe and tolerable in HD patients at doses of 100 mg and 200 mg per day over 8 weeks of use [Neurology 2004: 63:547-549].
The DOMINO study will assess the safety and tolerability of minocycline over a longer period of use by enrolling 100 subjects, age 18 or older with manifest HD, who will be followed approximately every three months for a total of 18 months. Please refer to the DOMINO Participating Site List for a site in your area, or the DOMINO Brochure (PDF file format) for more information.
TREND-HD: A Multi-Center, Double-Blind, Randomized, Placebo-Controlled Trial of Ethyl-EPA (Miraxion™) in Subjects with Mild to Moderate Huntington’s Disease
… Enrollment for TREND-HD has been completed as of August 2006 …
If you are interested in learning more about this study, please contact
the Huntington Study Group toll free at 1-800-487-7671.Please see the TREND-HD participating site list.
The Huntington Study Group (HSG) is conducting a study of the research medication ethyl-EPA in persons 35 years of age or older who have mild to moderate Huntington’s disease (HD). TREND-HD is designed to determine the effect of ethyl-EPA on the motor (movement) signs and symptoms of HD. In this 12-month study, 43 sites in the United States and Canada enrolled 316 research subjects with early signs of HD who are independently ambulatory (walking) and fully self-sufficient in activities of daily living, such as eating, dressing, and bathing. Enrollment began in the summer of 2005. The study is sponsored by Amarin Neurosciences in Scotland, U.K, a subsidiary of Amarin Corp., PLC in London.
For further information, please see the June 2005 Press Release and the April 2007 Press Release.
PHEND-HD: Phenylbutyrate Development for Huntington’s Disease - Multi-center, Double-Blind, Placebo-Controlled Study with Open-Label Follow-Up to Determine the Safety & Tolerability of Phenylbutyrate in Subjects with Huntington’s Disease
...Recruitment for the PHEND-HD study has been completed as of January 2006...The Huntington Study Group (HSG), under the direction of Steven M. Hersch, MD, PhD (Massachusetts General Hospital) and Karl Kieburtz, MD, MPH (University of Rochester) is conducting a multi-center, double-blind, placebo-controlled study with open-label follow-up of phenylbutyrate. PHEND-HD is designed to assess and gather information on the safety and tolerability of phenylbutyrate. This study recruited subjects who are 18 years of age or older with a clinical diagnosis of HD. The study will last approximately 20 weeks for each subject.
Participating sites include:
For further information on the study, contact:
Steven M. Hersch, MD, PhD
Principal Investigator
Massachusetts General Hospital
Boston, MA 02129
(617) 726-5892Huntington Study Group
University of Rochester
Rochester, NY 14620
(800) 487-7671
Pilot Safety and Tolerability Study of Coenzyme Q10 in Huntington's Disease and in Normal Subjects
...Recruitment for the Coenzyme Q10 study has been completed as of January 2004...
Please go to our Participating Site List for a site near you
The Huntington Study Group, under the direction of Karl Kieburtz, MD, MPH (University of Rochester), and Merit Cudkowicz MD, MSc (Massachusetts General Hospital), has conducted an open-label study to assess and gather information on the safety and tolerability of coenzyme Q10. This study has recruited participants who are 18 years of age or older, 20 with early manifest HD and also healthy persons without HD. Please see the CoQ10 study results.Participating sites include the Institute for Neurodegenerative Disorders, Massachusetts General Hospital, University of Rochester, and Wake Forest University.
TETRA-HD: A Randomized, Double-Blind, Placebo-Controlled, Study of Tetrabenazine for the Treatment of Huntington’s Chorea
...Recruitment for TETRA-HD has been completed as of December 2003...
Please see the TETRA-HD Participating Site List, the February 2006 TETRA-HD Press Release, and the August 2003 TETRA-HD Press Release.
The Huntington Study Group (HSG), under the direction of Frederick Marshall, MD (University of Rochester), has conducted a multi-center, double-blind, placebo-controlled study of tetrabenazine. TETRA-HD is designed to assess and gather information on the optimal dosage of tetrabenazine. The HSG has recruited more than 70 participants who are 18 years of age or older and who have chorea as a significant feature of their HD. For each patient the study will last approximately 15 weeks. At the end of the trial, patients will be offered the option to continue treatment for an additional 6 to 12 months. Please see the results of the TETRA-HD study.
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