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Huntington Study Group (presenter: C. Hyson, University of Rochester, Rochester, New York).

Background: Coenzyme Q10 (CoQ10), a compound with potential neuroprotective properties, was investigated at 600mg/day in HD patients and demonstrated a favorable, though not statistically significant, trend toward slowing disease progression. Higher doses of CoQ10 may prove beneficial.

Objective: To investigate the tolerability and plasma levels attained when administering 1200, 2400 and 3600 mg/day of CoQ10 to HD patients and healthy subjects.

Methods: Subjects enrolled in a 20-week open-label trial, starting on 1200 mg/day of CoQ10, increasing incrementally every 4 weeks by 1200 mg to a maximum dosage of 3600 mg/day. Monthly evaluations included review of adverse events and CoQ10 plasma levels. A 16 week extension phase for the HD subjects was carried out, using two other proprietary formulations of CoQ10 for 8 weeks each, at a dose of 3600mg/day.

Results: 28 subjects (20 HD, 8 healthy) were enrolled. 23 subjects (82%) achieved the target dosage of 3600mg/day. Six subjects (2 healthy, 4 HD) withdrew prematurely (gastrointestinal symptoms in 3, worsening HD in 2, and 1 due to a fall). All three serious adverse events (1 fall, 1 pathological fracture, and a malignancy) occurred in a single subject, and none were thought related to CoQ10. The most common adverse events were secondary to gastrointestinal symptoms. Mean blood levels achieved were: 1.26+1.27 mcg/ml (baseline), 5.59+2.24 mcg/ml (1200mg/day), 6.37+3.25 mcg/ml (2400mg/day), and 7.49+4.09 mcg/ml (3600mg/day). 16 subjects carried on in the extension phase. Mean blood levels at 3600mg/day with the other brands were 8.45+3.28 mcg/ml and 9.48+3.79 mcg/ml.

Conclusion: As a result of this study, the optimal dose of CoQ10 was felt to be 2400mg/day, as there was no substantial gain in blood levels at higher doses, but there were fewer adverse events at this dose.

Financial Disclosure: the authors do not have any personal financial interest in this research supported by the High Q Foundation.

E. Kayson(1), E. Julian-Baros(1), C. Weaver(1), A. Shinaman(1), C. Orme(1), J. Weber(1), S. Daigneault(1), A.B. Young(2), I. Shoulson(1) and the Huntington Study Group (HSG) PHAROS Investigators. (1)University of Rochester, Rochester New York, (2)Harvard University, Boston Massachusetts.

Objective: Recognizing effective recruitment techniques in an at-risk Huntington’s disease (HD) population will result in achievement of timely accrual goals for future studies.

Background: PHAROS is a longitudinal observational study to determine the earliest and most specific signs of HD in clinically unaffected adults at risk for HD. Literature exists for recruitment strategies in clinical trials. However, nothing is published examining an at-risk HD population that may have special confidentiality considerations.

Methods: Consenting men and women, 26-55 years, unaware of their genetic status, were recruited at 43 Huntington Study Group (HSG) research sites in the US and Canada. Information about PHAROS was communicated via the research center (site personnel, brochures, business cards), community education (support group presentations), and national awareness (press releases, call-in center, genetic counselors, mass mailings, website). Referral information was collected at enrollment.

Results: Recruitment of 1001 research participants was achieved in 4.5 years. Participants age 41.8 ± 7.3 years (mean ± sd) were predominately women (68.8%), Caucasian (98.1%), married (69.8%), highly educated (14.9 ± 2.6 years), and employed (95.7%). US sites recruited 875 (87.4%) participants with a geographic distribution predominately in the East and Canada recruited 126 (12.5%) participants. Sources of referrals were accounted for 715 PHAROS participants with 276 (38.6%) from PHAROS site personnel, 149 (20.8%) from family and friends, 82 (11.5%) from HD advocacy organizations, 75 (10.5%) from the National HD Research Roster, 61 (8.5%) from other sources, 31 (4.3%) from the HSG website, 11 (1.5%) from the HSG call-in center, and 30 (4.2%) from HD educational conferences.

Conclusions: Recruitment and evaluation of an at-risk population is an emerging area of research. These data show that the research sites and family and friends were the most effective sources of referrals. The disproportionate number of women enrolled may be unique for an at-risk group and may not be representative of the HD at-risk population as a whole.

Financial Disclosure: the authors do not have any personal financial interest in this research supported by the National Institutes of Health (HG 02449), the Hereditary Disease Foundation, the High Q Foundation and the Huntington’s Disease Society of America.

Huntington Study Group (presenter: F. Marshall, University of Rochester, Rochester, New York).

Objective: To examine the efficacy and dosing of tetrabenazine (TBZ) for treating chorea in Huntington disease (HD).

Background: TBZ selectively depletes central monoamines by reversibly binding to the type-2 vesicular monoamine transporter.

Methods: We randomized 84 patients with HD to receive TBZ (n=54) or placebo (n=30) for 12 weeks. Dosage, administered in 12.5 mg tablets, was increased over 7 weeks up to 8 tablets (100mg) daily, until the desired antichoreic effect or intolerable adverse effects occurred. The pre-specified primary outcome was the change from baseline in the chorea score of the Unified Huntington Disease Rating Scale (UHDRS). Additional outcomes included the Clinical Global Impression (CGI) scales, the UHDRS total motor score and gait, functional scales, tolerability, safety, and laboratory parameters.

Results: TBZ treatment resulted in a decline of 5.0 units in chorea score compared with a decline of 1.5 units on placebo treatment (adjusted mean effect size = -3.5 ± 0.8 UHDRS units (mean ± SE); 95% CI: -5.2, -1.9; p < 0.0001). TBZ was also superior to placebo as assessed by the CGI Global Improvement scale (0.75 units, p = 0.007), but not on UHDRS total motor score (p = 0.08). There were five withdrawals in the TBZ group and four serious adverse events in three subjects (drowning suicide, complicated fall, and restlessness/suicidal ideation), compared with one withdrawal in the placebo group. Forty-eight subjects (89%) in the TBZ group had at least one adverse effect, compared with 21 subjects (70%) in the placebo group (p = 0.03). No clinically significant alterations in surveillance laboratory tests were observed.

Conclusion: TBZ, at a dosage of up to 100 mg/day, is effective for the treatment of chorea in HD.

Financial Disclosure: the authors do not have any personal financial interest in this research supported by Prestwick Pharmaceuticals.

N.I. Mejia, C. Hunter, K. Flores, W.G. Ondo. Baylor College of Medicine, Houston, Texas.

Objective: To determine the clinical efficacy and safety of memantine in Huntington’s disease (HD).

Background: The excitatory activity of L-glutamate may affect the progression of HD. Memantine, an N-methyl-D-aspartate antagonist used to treat Alzheimer’s dementia, could theoretically retard the progression of this disorder, improve cognition, and improve chorea.

Methods: HD patients were recruited from Baylor College of Medicine; twelve were started on memantine, titrated to a daily dose of 20mg, and followed for three months. Other medications were unchanged during this period.

Results: Four patients stopped memantine because of lack of apparent efficacy (N= 2) and adverse events (N= 2). The eight analyzed patients: 4 female (50%), ages 58.8 years ± 14.8, with 44.7 ± 4.3 CAG repeats, titrated to the maximum dose of 20mg of memantine, and were followed for 3.6 ± 1.0 months. Although no significant difference existed between their initial (x= 10 ± 8.6) and final (x= 11.2 ± 9.4) total UHDRS motor scores (P= 0.159), patients had a significant (P= 0.009) decrease in their maximum chorea rating at their final visit (0.79 ± 0.51), compared to their initial evaluation (1.8 ± 0.8). Patients did not show a significant change in their cognitive (P= 0.899) or behavioral (P= 0.057) ratings. Their total functional capacity (P= 0.097) and independence scale rating (P= 0.842) also failed to show a significant change. Most (N= 6, 75%) patients did not have any adverse effects under memantine; one (12.5%) reported drowsiness; another (12.5%) complained of worsening balance, speech and social interaction. No serious adverse events were reported.

Conclusions: In this small pilot trial, 20mg daily dose of memantine significantly decreased chorea, but failed to show improvement in patient’s cognitive, behavioral, functional, or independence ratings. Most patients tolerated memantine without side effects. Larger controlled trials and long term trials to assess for disease modification are justified.

Financial Disclosure: None.

J.-L. Li(1), J.F. Gusella(2), M.E. MacDonald(2), R.H. Myers(1), for the investigators of the HD MAPS Study. (1)Department of Neurology, Boston University School of Medicine, Boston, Massachusetts; (2)Molecular Neurogenetics Unit, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.

Age at onset (AO) of Huntington’s disease (HD) is correlated with the size of the abnormal of CAG repeats expansion in the HD gene. However, several studies have indicated that other genetic factors also contribute to the variability in HD AO. To identify modifier genes, we recently reported a whole-genome scan in a sample of 629 affected sibling pairs from 295 pedigrees, in which six genomic regions provided suggestive evidence for quantitative trait loci (QTL) modifying AO in HD [Li et al. 2003]. In the present study, we further assessed evidence for linkage in 125 newly recruited sibling pairs from 57 pedigrees. Eighteen microsatellite markers, three from each of six loci, were genotyped for this study. Using a variance component method, we performed multipoint linkage analysis in the follow-up sample and the combined sample (352 pedigrees with 754 sibling pairs). Confirmation of linkage was observed at 6q23-24 in the follow-up sample (LOD = 2.39, p = 0.0005) and generated strong evidence for linkage in the combined sample (LOD = 5.10, p < 10-6). This region contains several interesting candidate genes, encoding proteins with established roles in cell death and nervous system development, including glutamate receptors, apoptosis effectors, transcription factors, and others. Studies to identify genetic modifiers for AO for HD are in progress.

Financial Disclosure: the authors do not have any personal financial interest in this research supported by the National Institute Health (NS16367-24).

Huntington Study Group (HSG) PHAROS Investigators (presenter: A. Shinaman, University of Rochester; Rochester, New York).

Objective: To determine the willingness of individuals at risk for Huntington’s Disease (HD) to consent for future unspecified research use of blood samples.

Background/Methods: Between July 1999-January 2004, 1001 research participants were enrolled in PHAROS to determine the early and gene-specific signs of HD. Participants consented to provide a blood sample for CAG HD gene analysis with the provision that individually identifying results would never be disclosed, and to the retention of a blood sample for future unspecified research related to HD.

Results: For the 990 participants who provided samples and instructions, 906 (91.5%) agreed to future unspecified research with the sample. Eighty-four (8.4%) participants asked for their sample to be destroyed at the conclusion of the PHAROS study. Neither gender, race nor ethnicity were factors in the decision. Of the 116 participants who were re-consented during the study due to changes to the protocol, 115 (99.1%) provided identical instructions regarding future use of their samples.

Conclusions: Individuals at risk for HD are overwhelmingly willing to consent to future unspecified HD research using their stored blood sample. These results are in keeping with other studies, particularly where research may provide new knowledge about a disease affecting research participants.

Financial Disclosure: the authors do not have any personal financial interest in this research supported by the National Institutes of Health (HG 02449), the Hereditary Disease Foundation, the High Q Foundation and the Huntington’s Disease Society of America.

Depression and Suicidality at Baseline in the Prospective Huntington At Risk Observational Study (PHAROS)

Author list: Kayson E(1), Darnell M(1), Weber J(1), Biglan K(2), Shoulson I(1) and the Huntington Study Group PHAROS Investigators--see the complete author list below the abstract

Institutions: (1)University of Rochester, Rochester NY; (2)Johns Hopkins School of Medicine, Baltimore, MD

Objective: PHAROS aims to identify the early and gene-specific signs of clinical onset in unaffected adults at risk for Huntington’s Disease (HD) who have chosen not to undergo presymptomatic DNA testing but have consented to participate in this observational research to assess the feasibility and develop the methodology for preventative clinical trials.

Background: Depression occurs in about 40% of HD patients. The prevalence of completed suicide is 4.0 % – 7.3% in manifest HD and 1.8% – 5.3% among individuals at 50% risk. Depression and suicidal behavior have not been systematically assessed in individuals at risk for HD.

Methods: At enrollment, we obtained histories of depression and suicidal behavior and measured the Beck Depression Inventory (BDI) I or II, Beck Hopelessness Scale and the Unified Huntington’s Disease Rating Scale (UHDRS) in the research participants who consented to participate in PHAROS.

Results: Among the 1001 research participants (689 women, 312 men), a history of depression was reported by 286 (229 women, 57 men), suicidal ideation by 94 (74 women, 20 men), suicide attempts by 39 (34 women, 5 men), and 150 (117 women, 33 men) reported taking antidepressants. Among the 924 participants who had normal BDI scores, 239 (25.9%) reported a history of depression, 76 (8.2%) suicidal ideation, 32 (3.5%) suicide attempts, and 120 (13.0%) reported taking antidepressants; among the 65 participants who had BDI scores in the depressive range, 41 (63.1%) reported a history of depression, 16 (24.6%) suicidal ideation, 7 (10.8%) suicide attempts, and 28 (43.1%) reported taking antidepressants.

Conclusions: Reports of depression, and suicidal behavior at baseline in the PHAROS cohort were more common in our female than male participants and among those with BDI measures in the depressive range. Depression and adverse life events, such as the prospects for developing HD, likely predispose to suicidal behavior. Identification of these and other risk factors will help protect the well being of the PHAROS cohort and other individuals participating in clinical trials aimed at postponing the onset of HD.

Financial Disclosure: The authors do not have any personal financial interest in this research, which is supported by the National Institutes of Health (HG 02449), the Hereditary Disease Foundation, the High Q Foundation, and the Huntington’s Disease Society of America.

Prospective Huntington At-Risk Observational Study (PHAROS)
PHAROS Investigators for the Huntington Study Group

Steering Committee: University of Rochester, Rochester, NY, Ira Shoulson, MD (principal investigator); Massachusetts General Hospital, Charleston, MA, Anne Young, MD, PhD (co-principal investigator); University of Rochester, Rochester, NY, Karl Kieburtz, MD, MPH (Director, Clinical Trials Coordination Center); David Oakes (chief biostatistician); Elise Kayson, MS, RNC (project coordinator); Hongwei Zhao, PhD, M. Aileen Shinaman, JD (HSG executive director); Johns Hopkins University, Baltimore, MD, Kevin Biglan, MD (medical monitor); Massachusetts General Hospital, Charleston, MA, Steven Hersch, MD, PHD, Jack Penney, MD (deceased), Columbia University Medical Center, New York, NY, Karen Marder, MD; University of Iowa, Iowa City, IA, Jane Paulsen, PhD; Indiana University School of Medicine, Indianapolis, IN, Kimberly Quaid, PhD; Lily Corporate Center, Indianapolis, IN, Eric Siemers, MD; The Parkinson’s Institute, Sunnyvale, CA, Caroline Tanner, MD

Event Monitoring Committee: Massachusetts General Hospital, Charleston, MA: Steven Hersch, MD, PhD (co-chair); Indiana University, Bloomington, IN: Julie Stout, PhD (co-chair); University of Iowa, Iowa City, IA: William Coryell, MD, Cheryl Erwin, PhD; Wake Forest University School of Medicine, Winston-Salem, NC: Vicki Hunt, RN; Johns Hopkins University, Baltimore, MD: Christopher Ross, MD, PhD; Minnesota Center for Health Care Ethics, Minneapolis, MN; Dorothy Vawter, PhD; one advocacy organization representative

Independent Monitoring Committee: Columbia University Medical Center, New York City, NY: Stanley Fahn, MD (Chair); Weiu-Yann Tsai, PhD; Indiana University Medical Center, Indianapolis, IN: Michael Conneally, PhD

Participating Investigators and Coordinators: Hereditary Neurological Disease Centre (HNDC), Wichita, KS: William Mallonee, MD, David Palmer, MD, Greg Suter, BA; University of Kansas Medical Center, Kansas City, KS: Richard Dubinsky, MD, Gary Gronseth, MD, R. Neil Schimke, MD, Carolyn Gray, RN; Hennepin County Medical Center/Minneapolis, Minneapolis, MN: Martha Nance, MD, Scott Bundlie, MD, Dawn Radtke, RN; Ohio State University, Columbus, OH: Sandra Kostyk, MD, PhD, George W. Paulson, MD, Karen Thomas, DO, Nonna Stepanov, MD; Wake Forest University School of Medicine, Winston-Salem, NC: James Caress, MD, Francis Walker, MD, Vicki Hunt, RN; Hotel-Dieu Hospital-CHUM, Montreal, QC: Sylvain Chouinard, MD, Guy Rouleau, MD, PhD, Hubert Poiffaut, RN; Emory University School of Medicine, Atlanta, GA: Claudia Testa, MD, PhD, Timothy Greenamyre, MD, PhD, Joan Harrison, RN; University of California, San Diego (UCSD), LaJolla, CA: Jody Corey-Bloom, MD, PhD, David Song, MD, Guerry Peavy, PhD, Jody Goldstein, BS; University of Iowa, Iowa City, IA: Jane Paulsen, PhD, Henry Paulson, MD, Robert L. Rodnitzky, MD, Ania Mikos, BA; Columbia University Medical Center, New York, NY: Karen Marder, MD, PhD, Elan Louis, MD, Carol Moskowitz, RN; Indiana University School of Medicine, Indianapolis, IN: Kimberly Quaid, PhD, Joanne Wojcieszek, MD, Melissa Wesson, MS; University of Washington & VA Puget Sound Health Care System, Seattle, WA: Ali Samii, MD, Thomas Bird, MD, Hillary Lipe, ARNP; Medical College of Wisconsin, Milwaukee, WI: Norman Reynolds, MD, Karen Blindauer, MD, Jeannine Petit, ANP; University of Rochester, Rochester, NY: Peter Como, PhD, Frederick Marshall, MD, Carol Zimmerman, RN; Oregon Health & Science University, Portland, OR: Penelope Hogarth, MD, John Nutt, MD, Pamela Andrews, BS, CCRC; Massachusetts General Hospital, Charleston, MA: Steven Hersch, MD, PhD, Diana Rosas, MD, Yoshio Kaneko, BA, Sona Gevorkian, MS; Mayo Clinic Scottsdale, Scottsdale, AZ: John Caviness, MD, Charles Adler, MD, PhD, Susan Bernstein, RN, CRC; University of California Davis, Sacramento, CA: Vicki Wheelock, MD, David Richman, MD, Terry Tempkin, RNC, MSN; Brown University (Memorial Hospital of Rhode Island), Pawtucket, RI: Chuang-Kuo Wu, MD, Joseph H. Friedman, MD, Margaret Lannon, RN, MS; Colorado Neurological Institute, Englewood, CO: Lauren Seeberger, MD, Rajeev Kumar, MD, Robin Dorsey, BA, CCRC; University of Michigan, Ann Arbor, MI: Ninith Kartha, MD, Roger Albin, MD, PhD, Kristine Wernette, RN, MS; Washington University, St. Louis, MO: Brad Racette, MD, Joel S. Perlmutter, MD, Laura Good, BA; UCLA Medical Center, Los Angeles, CA: George Jackson, MD, PhD, Susan Perlman, MD, Shelley Segal; University of Alberta, Edmonton, Alberta: Wayne Martin, MD, Ted Roberts, MD, Marguerite Wieler, BSC, PT; University of British Columbia, Vancouver, British Columbia: Blair Leavitt, MD, Lynn Raymond, MD, PhD, Joji Decolongon, MSC; Baylor College of Medicine, Houston, TX: William Ondo, MD, Joseph Jankovic, MD, Kevin Dat Nguyen-Vuong, MA; University of South Florida, Tampa, FL: Robert Hauser, MD, Juan Sanchez-Ramos, MD, PhD, Karen Price, MA; University of Calgary, Calgary, Alberta: Sarah Furtado, MD, PhD, Oksana Suchowersky, MD, Mary Lou Klimek, RN, MA; Centre for Addiction and Mental Health, Markham, Ontario: Rustom Sethna, MD, Mark Guttman, MD, Sandra Russell, BSW, RSW; North Shore University Hospital, Manhasset, NY: Marc Mentis, MB, CHB, Andrew Feigin, MD, Marie Cox, RN, BSN; University of Alabama at Birmingham, Birmingham, AL: Alan Percy, MD, Leon Dure, MD, Donna Pendley, RN; University of Virginia, Charlottesville, VA: Madaline Harrison, MD, Frederick Wooten, MD, Elke Rost-Ruffner, RN, BSN; UMDNJ Robert Wood Johnson Medical Center, Stratford, NJ: William Johnson, MD, Gerald Podskalny, DO, Lisa Giffin, LPN; University of Pennsylvania, Philadelphia, PA: Amy Colcher, MD, Andrew Siderowf, MD, Mary Matthews, RN; Institute for Neurodegenerative Disorders, New Haven, CT: Danna Jennings, MD, Kenneth Marek, MD, Karen Caplan, MSW; Albany Medical College, Albany, NY: Stewart Factor, DO, Donald Higgins, MD, Eric Molho, MD, Constance Nickerson, LPN; Winnipeg Clinic, Winnipeg, Manitoba: Douglas Hobson, MD, Paul Shelton, MD, Shaun Hobson, RN; University of Miami, Miami, FL: Carlos Singer, MD, Nestor Galvez-Jimenez, MD; Doris Martin; Rush Presbyterian-St. Luke’s Medical Center, Chicago, IL: Kathleen Shannon, MD, Cynthia Comella, MD, Jeana Jaglin, RN, CCRC; University of Maryland School of Medicine, Baltimore, MD: Karen Anderson, MD, William Weiner, MD, Kelly Dustin, RN, BSN; Johns Hopkins University, Baltimore, MD: Adam Rosenblatt, MD, Christopher Ross, MD, PhD, Deborah Pollard; Boston University, Boston, MA: Marie H. Saint-Hilaire, MD, Peter Novak, MD, Melissa Diggin, MS, RN; University of Connecticut, Hartford, CT: Wallace Deckel, PhD, James Duffy, MD, Mary Jane Fitzpatrick, APRN.

Biostatistics and Clinical Trials Coordination Center: University of Rochester: Alicia Brocht, BA, Susan Daigneault, Megan Darnell, MS, Karen Gerwitz, BS, Connie Orme, BA, Victoria Ross, MA, Mary Slough, Arthur Watts, BS, Joseph Weber, BS, Christine Weaver, Hongwei Zhao, PhD

Consultant: Massachusetts General Hospital: Marcy MacDonald, PhD

Financial Disclosure: All compensation received by investigators for research services was paid through a contract between the University of Rochester and the sponsors.

Acknowledgment: We thank our 43 PHAROS Sites, the participants in this study and the National Research Roster for HD Patients and Families.

Funding/Support: PHAROS is primarily supported by the National Human Genome Research Institute (NHGRI) and the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (HG 02449). PHAROS is also supported by the Huntington’s Disease Society of America, the Huntington Society of Canada, the Hereditary Disease Foundation, and the High Q Foundation.

A Randomized, Double-blind, Placebo-Controlled Study of Tetrabenazine in Patients with Huntington’s Disease

Authors: Huntington Study Group--see the complete author list below the abstract

Objective: To establish the efficacy of tetrabenazine (TBZ) for treating chorea in Huntington disease (HD).

Background: TBZ selectively depletes central monoamines by reversibly binding to the type-2 vesicular monoamine transporter.

Methods: We randomized 84 HD subjects to TBZ 12.5mg tablets (n=54) or placebo (n=30) for 12 weeks. Dosage was increased over 7 weeks up to 8 tablets (100mg) daily, until the desired antichoreic effect or intolerable side effects occurred. The primary outcome was the change from baseline in Unified Huntington Disease Rating Scale (UHDRS) chorea score. Secondary outcomes included the Clinical Global Impression (CGI), the UHDRS total motor score, functional scales, tolerability, safety, and laboratory parameters.

Results: The adjusted mean TBZ chorea score declined by 5.0 UHDRS units, while placebo declined by 1.5 units (p < 0.0001). TBZ was also superior to placebo assessed by the CGI scale (0.75 units, p = 0.007), but showed no significant impact on UHDRS total motor score (p = 0.08). The impact of TBZ on functional scales was explored. There were five withdrawals in the TBZ group and four serious adverse events in three subjects (drowning death, complicated fall, and restlessness/suicidal ideation) compared to one withdrawal in the placebo group. Forty-eight subjects (89%) in the TBZ group had at least one adverse effect, compared with 21 subjects (70%) in the placebo group (p = 0.03). Laboratory measures and vital signs were unchanged.

Conclusions: Tetrabenazine, adjusted to a dosage of up to 100 mg/day, is effective for the treatment of chorea in HD.

Steering Committee: Frederick J. Marshall, MD (principal investigator, University of Rochester School of Medicine and Dentistry); Francis Walker, MD (co-principal investigator, Wake Forest University School of Medicine), Samuel Frank, MD (medical monitor, Boston University School of Medicine); David Oakes, PhD (chief biostatistician); Sandra Plumb, BS (chief study coordinator); Ira Shoulson, MD; Aileen Shinaman, JD (University of Rochester School of Medicine and Dentistry); Stewart A Factor, DO (Albany Medical Center); Stanley Fahn, MD (Columbia-Presbyterian Medical Center, Neurological Institute); Victoria P. Hunt, RN (Wake Forest University School of Medicine); Joseph Jankovic, MD (Baylor College of Medicine).

Site Coordinators/Investigators: Jody Goldstein, BS; Jody Corey-Bloom, MD (University of California, San Diego); Joann Belden, RN; Joanne Wojcieszek, MD (Indiana University School of Medicine); Constance Nickerson, LPN; Donald Higgins, MD (Albany Medical College); Marcia McCall, MS; Juan Sanchez-Ramos, MD (University of South Florida); Nonna Stepanov, MD; Sandra Kostyk, MD, PhD (Ohio State University); Joan Harrison, RN; Claudia Testa, MD (Emory University School of Medicine); Barbara Shannon, RN; Andrew Feigin, MD (North Shore University Hospital); Dawn Radtke, RN; Martha Nance, MD (University of Minnesota/Hennepin County Medical Center); Penny Stanton, CCRP; Tetsuo Ashizawa, MD (University of Texas Medical Branch); Christine Hunter, RN; William Ondo, MD (Baylor College of Medicine); Katherine Rose, BA; Michael Geschwind, MD, PhD (University of California, San Francisco); Jeana Jaglin, RN; Kathleen Shannon, MD (Rush-Presbyterian-St. Luke’s Medical Center); Mary Matthews, RN; Amy Colcher, MD (University of Pennsylvania); Pam Riendl, RN, MSN; Karen Blindauer, MD (Medical College of Wisconsin); Kelly Dustin, RN; Karen Anderson, MD (University of Maryland School of Medicine); Susan Fowler, RN, PhD; Deviyani Mehta, MD; Philip Hanna, MD (New Jersey Neuroscience Institute).

Coordination Center and Biostatistics: Patricia Lindsay, Shirley Eberly, PhD; Hongwei Zhao, ScD. (University of Rochester School of Medicine and Dentistry)

DNA Testing Center: Marcy MacDonald, PhD. (Massachusetts General Hospital, Charlestown Labs)

Prestwick Pharmaceuticals Authors: Kathleen Clarence-Smith, MD, PhD; Christopher O’Brien, MD; Lucinda Wilson.

We thank the following individuals for their contributions to the conduct of this study:
Jan Bausch; Stephen Bean, RPH; Sue Henderson, Peggy Lamberton, Letty Laskowski, Margot Lutz, RN, Karen Rothenburgh, Earl Westerlund (University of Rochester); Peggy Laird; Ben Lewis, PhD; Arthur Watts, BS; Marion Yonce, MS; Kathryn Zunich, MD (Prestwick Pharmaceuticals).

Clinical Research Consultants: Amy T. Fletcher (Milan, IN); Timothy D. Foster (College Station, TX); Daniel A. Rooney, JR, BS (Portland, OR); Kathleen Karthaeuser (Sterling, NJ).

We gratefully acknowledge the members of the Independent Data and Safety Monitoring Committee for this trial: Anthony Lang, MD (chair) (University of Toronto); Michael McDermott, PhD; Pierre Tariot, MD; Saleem Ishmael, MD (University of Rochester).

This study was funded by a grant from Prestwick Pharmaceuticals, Inc. to the University of Rochester and in turn through subcontracts to the participating research sites. The Huntington Study Group (HSG) is a non-profit consortium of HD investigators. None of the HSG investigators or staff had any personal financial relationship with Prestwick Pharmaceuticals, Inc, either in the form of equity interests or consulting fees. The HSG Coordination and Biostatistics Centers at the University of Rochester independently compiled and analyzed the data for this study.

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