Huntington DiseasePHAROSNCT00052143
Prospective Huntington At Risk Observational Study
An observational study of Huntington Disease, run by Huntington Study Group at 42 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 26 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Huntington Disease
- How many people
- 1,001 participants
- Where
- 42 sitesin 2 countries
- Study dates
- 9 Jul 1999to 17 Dec 2009
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 26 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Study population: HD Community
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
42 sites in 2 countries.
42 sites in 2 countries
United States37 sites
Albany Medical College
Albany, New York
University of Michigan
Ann Arbor, Michigan
Emory University
Atlanta, Georgia
Johns Hopkins University
Baltimore, Maryland
University of Maryland at Baltimore
Baltimore, Maryland
University of Alabama
Birmingham, Alabama
Boston University School of Medicine
Boston, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts
University of Virginia
Charlottesville, Virginia
Rush University Medical Center
Chicago, Illinois
Ohio State University
Columbus, Ohio
Colorado Neurological Institute
Englewood, Colorado
University of Connecticut
Hartford, Connecticut
Baylor College of Medicine
Houston, Texas
Indiana University School of Medicine
Indianapolis, Indiana
University of Iowa
Iowa City, Iowa
University of Kansas Medical Center
Kansas City, Kansas
University of California at San Diego
La Jolla, California
UCLA School of Medicine
Los Angeles, California
North Shore-LIJ Health System
Manhasset, New York
University of Miami School of Medicine
Miami, Florida
Medical College of Wisconsin-Milwaukee
Milwaukee, Wisconsin
Hennepin County Medical Center
Minneapolis, Minnesota
Institute for Neurodegenerative Disorders
New Haven, Connecticut
Columbia University
New York, New York
NeuroHealth Parkinson's Disease Movement Disorders Center
Pawtucket, Rhode Island
University of Pennsylvania
Philadelphia, Pennsylvania
Mayo Clinic Arizona
Phoenix, Arizona
Oregon Health and Science University
Portland, Oregon
University of Rochester
Rochester, New York
University of California, Davis
Sacramento, California
University of Washington and VA Puget Sound
Seattle, Washington
Washington University School of Medicine
St Louis, Missouri
UMDNJ Huntington Disease Service Center
Stratford, New Jersey
University of South Florida
Tampa, Florida
Hereditary Neurological Disease Center
Wichita, Kansas
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina
Canada5 sites
University of Calgary
Calgary, Alberta
University of Alberta
Edmonton, Alberta
The Centre for Addiction and Mental Health
Markham, Ontario
Hotel-Dieu Hospital-CHUM
Montreal, Quebec
University of British Columbia
Vancouver, British Columbia
About this study
Official title: Prospective Huntington At Risk Observational Study (PHAROS)
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.
Read the 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.
- Conditions
- Huntington Disease
- Keywords
- Huntington's disease, HD, Huntington disease
- Medical subject headings
- Huntington Disease
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2003 (estimated)
- Study start9 Jul 1999
- Primary completion17 Dec 2009
- Study completion17 Dec 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,001 participants (actual)
Sponsor and investigators
- Lead sponsor
- Huntington Study Group
- Sponsor type
- Research network
- Collaborators
- National Human Genome Research Institute (NHGRI), National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Sponsor
- Investigators
- Ira Shoulson, M.D., Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00052143
- Sponsor's protocol ID
- R01HG002449
- Acronym
- PHAROS
- Status last verified
- Aug 2024
- First submitted
- 23 Jan 2003
- Last updated
- 3 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 16831969Oster 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.
PubMed 18627059
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Huntington At Risk Observational Study (PHAROS)
- ClinicalTrials.gov ID
- NCT00052143
- Acronym / sponsor ID
- PHAROS / R01HG002449
- Conditions
- Huntington Disease
- Keywords
- Huntington's disease, HD, Huntington disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,001 participants (actual)
- Lead sponsor
- Huntington Study GroupResearch network
- Collaborators
- National Human Genome Research Institute (NHGRI), National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- 9 Jul 1999 (actual)
- Primary completion
- 17 Dec 2009 (actual)
- Study completion
- 17 Dec 2009 (actual)
- First posted
- 24 Jan 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 26 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
- Study population
- HD Community
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2024.
- Background: PubMed 16831969Huntington 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.
- Derived from this study: PubMed 18627059Oster 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.
- study websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ira Shoulson, M.D. principal investigator
University of Rochester
42 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Huntington Disease, run by Huntington Study Group at 42 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 42 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00052143, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2024.