Clinical trial NCT00052143: Prospective Huntington At Risk Observational Study
Official title: Prospective Huntington At Risk Observational Study (PHAROS)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Huntington Disease
Keywords: Huntington's disease
HD
Huntington disease
Lead sponsor: Huntington Study Group
Sponsor class: NETWORK
Collaborators: name: National Human Genome Research Institute (NHGRI)
name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2003-01-24
Enrollment: 1001
Sex: ALL
Minimum age (years): 26
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: HD Community

Brief summary: The purpose of this study is to define the natural history and experiences of people who are at risk for developing Huntington's disease but who do not know their genetic status.

Detailed description: Huntington's disease (HD) is a genetic disease characterized by changes in movement and behavior. To date, little research has been done on individuals who are at risk for developing the disease. PHAROS is an observational study designed to monitor people who are at risk for developing HD, and to survey their attitudes and beliefs about their "at-risk" status. Investigators hope this study will help answer some important questions about HD, such as: 1.) What are the earliest signs of HD and when do they start? 2.) How accurate are the measures that physicians use in detecting the onset of HD? 3.) What factors influence the age at which a person carrying the HD gene develops the illness? and 4.) In a group of people at risk for HD, how many will develop signs of the illness over a minimum three-year period of observation?

Participants in the study will be evaluated every 9 months (for 5 years) using the Unified Huntington's Disease Rating Scale (UHDRS)--a clinical tool which looks at movement, psychological and behavioral function. The participants will also be asked to complete surveys about their mood, life events, and attitudes or beliefs about being at risk for HD. A blood sample, taken at the beginning of the study, will be confidentially tested to determine if the participant has the abnormal gene for HD. Neither the participant nor any of the PHAROS physicians or coordinators will learn the results of individual gene tests.

The scientists hope that this study will provide essential information for future trials of experimental drugs for HD.

Eligibility criteria: Inclusion:

* Men and women between 26-55 years of age;
* Who are at risk for HD by virtue of having (or had) a parent or sibling with the illness;
* Who have never been tested for the HD gene, and who do not desire to be tested for the HD gene during the duration of the study;
* Agree to blinded (sample will be coded) genetic testing of the HD gene; and
* Who have never been diagnosed with HD.

Exclusion:

Individuals Who:

* have been diagnosed with HD;
* are taking antipsychotic medications;
* use antiemetic (anti-nausea) medications with on a regular basis (greater than 3 times per month); or
* exhibit clinical signs/symptoms of psychosis at the screening visit

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Phoenix
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: La Jolla
country: United States
facility: University of California at San Diego
state: California
city: Los Angeles
country: United States
facility: UCLA School of Medicine
state: California
city: Sacramento
country: United States
facility: University of California, Davis
state: California
city: Englewood
country: United States
facility: Colorado Neurological Institute
state: Colorado
city: Hartford
country: United States
facility: University of Connecticut
state: Connecticut
city: New Haven
country: United States
facility: Institute for Neurodegenerative Disorders
state: Connecticut
city: Miami
country: United States
facility: University of Miami School of Medicine
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Wichita
country: United States
facility: Hereditary Neurological Disease Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Baltimore
country: United States
facility: University of Maryland at Baltimore
state: Maryland
city: Boston
country: United States
facility: Boston University School of Medicine
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Minneapolis
country: United States
facility: Hennepin County Medical Center
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Stratford
country: United States
facility: UMDNJ Huntington Disease Service Center
state: New Jersey
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: Manhasset
country: United States
facility: North Shore-LIJ Health System
state: New York
city: New York
country: United States
facility: Columbia University
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest University Baptist Medical Center
state: North Carolina
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pawtucket
country: United States
facility: NeuroHealth Parkinson's Disease Movement Disorders Center
state: Rhode Island
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Seattle
country: United States
facility: University of Washington and VA Puget Sound
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin-Milwaukee
state: Wisconsin
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Vancouver
country: Canada
facility: University of British Columbia
state: British Columbia
city: Markham
country: Canada
facility: The Centre for Addiction and Mental Health
state: Ontario
city: Montreal
country: Canada
facility: Hotel-Dieu Hospital-CHUM
state: Quebec
Study officials:
affiliation: University of Rochester
name: Ira Shoulson, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Huntington Study Group PHAROS Investigators. At risk for Huntington disease: The PHAROS (Prospective Huntington At Risk Observational Study) cohort enrolled. Arch Neurol. 2006 Jul;63(7):991-6. doi: 10.1001/archneur.63.7.991.
citation: Oster E, Dorsey ER, Bausch J, Shinaman A, Kayson E, Oakes D, Shoulson I, Quaid K; Huntington Study Group PHAROS Investigators. Fear of health insurance loss among individuals at risk for Huntington disease. Am J Med Genet A. 2008 Aug 15;146A(16):2070-7. doi: 10.1002/ajmg.a.32422.