PHAROS

Prospective Huntington At Risk Observational Study

PHAROS completed enrollment of 1001 research participants in January 2004. The study is expected to conclude in 2010. HSG sites participating in PHAROS are listed below.

What is the PHAROS Project?

PHAROS aims to define the natural history and experiences of adults who are at risk for developing HD and who choose not to learn their gene status.

The PHAROS project is an observational study in North America (US and Canada) of people ages 26-55 who are at risk for Huntington Disease and have chosen not to learn of their HD gene status through predictive DNA testing. Research participants have been evaluated about every nine (9) months, now for an average of about 5 years, using a variety of clinical tests and surveys examining movement, psychosocial and behavioral function.

A research blood sample was obtained at the beginning of the study to examine the HD and related genes. No one, including the investigators and research participants will ever learn about the individual results of these gene analyses. Samples are also being collected to look for biomarkers of DNA and RNA damage.

Why is the HSG observing people at risk for Huntington Disease?

To date, little long-term (longitudinal) research has been done on individuals who are at risk for developing an inherited disease (such as a person who has a parent or sibling affected by HD). We hope this study will help us to answer some important questions, such as:

What are the earliest clinical signs of HD and when do they start?
How accurate are these measures in detecting the onset of HD?
What factors influence when a person carrying the HD gene develops signs and symptoms?
In a group of people at risk for HD, how many will develop signs of the illness over the multi-year period of observation?

We hope that this study may provide some essential information for future trials of experimental drugs for HD.

Who is enrolled in PHAROS?

Individuals:
Between 26-55 years of age;
Who are at risk for HD by virtue of having (or having had) a parent or sibling with the illness;
Who had never been tested for the HD gene, and who did not desire to be tested for the HD gene during the duration of the study; and
Who had never been diagnosed with HD.

How is confidentiality maintained in PHAROS?

Confidentiality is a central concern in the PHAROS study, and we have designed the study to protect the confidentiality of research subjects to the fullest extent possible. Names do not appear on the study forms - instead, a code number is assigned to identify research participants. A bar code (different from the subject code) is used to identify blood samples and any DNA results.

As mentioned previously, DNA results will not be provided to research participants or any of the physicians or staff at the site where the study is being conducted - instead, data will be transformed and sealed in a coded fashion (which does not identify individuals by name) and sent to a central secure location at the University of Rochester.

Could eligible persons participate if they did not want to learn their DNA results or whether they might be developing signs of HD?

Yes. Participants will never learn their DNA results through the PHAROS study, even when the study is complete. Participants can, if they later choose, arrange for DNA testing at an independent laboratory outside of the study. Research participants will also not be told if they are developing signs of HD. (If participants decide they do want to know this information, they will need to arrange for a clinical evaluation outside of the scheduled PHAROS study visits.)

Are there any risks to PHAROS participants?

Some psychological stress may occur from participation in PHAROS due to concern about showing signs of HD or carrying the HD gene. There are also some minor risks when blood is drawn for the blood sample. These risks are further explained in the consent form, and the research investigator or coordinator can also answer any questions.

What is the benefit of participating in PHAROS?

There is no direct health benefit from participating in PHAROS. However, participation may help to provide information useful for a better understanding of the onset of illness in unaffected persons at risk for HD.

How do I find out more information about the PHAROS Study?

As of January 2004, PHAROS completed enrollment. However, if you are interested in learning more about this study, please read our published articles.

For a participating site near you, please go to our PHAROS Participating Site List.

For access to the PHAROS Participant Newsletters, please see PHAROS Newsletter Volume Listing below.

PHAROS UPDATE - July 28, 2006

At Risk for Huntington Disease.
The PHAROS (Prospective Huntington At Risk Observational Study)
Cohort Enrolled
The Huntington Study Group PHAROS Investigators

The Huntington Study Group (HSG) PHAROS (Prospective Huntington At Risk Observational Study) investigators reported on the baseline characteristics of this remarkable cohort in the July 2006 issue of the Archives of Neurology (63:991-998). PHAROS is a multi-site, multi-year project that aims to gain knowledge about the early signs of Huntington disease (HD) onset and their relationship to environmental and genetic factors. PHAROS is also examining the psychosocial, ethical and practical issues involved in long-term study of adults at 50:50 risk to develop HD who have chosen not to undergo predictive genetic testing to learn of their gene-carrier status.

The PHAROS cohort of 1001 adults at risk for HD is mostly women (65%), well educated, and gainfully employed. At the start of the study, enrolled research participants were characterized as highly functional with minimal, if any, impairment of movement or intellect. Participants have enrolled in PHAROS mindful that the knowledge from this long-term study will help shape the efficient design and appropriate conduct of future clinical trials aimed at delaying the onset of illness in gene carriers who have not yet developed signs of HD. The knowledge from PHAROS will also inform us about how persons at high risk to develop a disabling genetic disease deal with lingering uncertainties about their future health and complex choices about their participation in research.

last updated: August 2009


Home Clinical Research Clinical Trials/Observational Studies in Progress 2CARE COHORT CREST-E DIMOND HART HORIZON PHAROS PREDICT-HD PREQUEL RESPOND-HD Completed Clinical Trials HSG Research Sites Request for Proposals About Us HSG Overview HSG Executive Committee HSG Working Groups Standing HSG Committees Becoming an HSG Research Site Education Resources Login FAQ's Clinical Care UHDRS Links Publications News & Events Events & Upcoming Meetings PSG/HSG Symposium Huntington Disease Clinical Research Symposium Giving Contact Us	Prospective Huntington At Risk Observational Study PHAROS completed enrollment of 1001 research participants in January 2004. The study is expected to conclude in 2010. HSG sites participating in PHAROS are listed below. What is the PHAROS Project? PHAROS aims to define the natural history and experiences of adults who are at risk for developing HD and who choose not to learn their gene status. The PHAROS project is an observational study in North America (US and Canada) of people ages 26-55 who are at risk for Huntington Disease and have chosen not to learn of their HD gene status through predictive DNA testing. Research participants have been evaluated about every nine (9) months, now for an average of about 5 years, using a variety of clinical tests and surveys examining movement, psychosocial and behavioral function. A research blood sample was obtained at the beginning of the study to examine the HD and related genes. No one, including the investigators and research participants will ever learn about the individual results of these gene analyses. Samples are also being collected to look for biomarkers of DNA and RNA damage. Why is the HSG observing people at risk for Huntington Disease? To date, little long-term (longitudinal) research has been done on individuals who are at risk for developing an inherited disease (such as a person who has a parent or sibling affected by HD). We hope this study will help us to answer some important questions, such as: What are the earliest clinical signs of HD and when do they start? How accurate are these measures in detecting the onset of HD? What factors influence when a person carrying the HD gene develops signs and symptoms? In a group of people at risk for HD, how many will develop signs of the illness over the multi-year period of observation? We hope that this study may provide some essential information for future trials of experimental drugs for HD. Who is enrolled in PHAROS? Individuals: Between 26-55 years of age; Who are at risk for HD by virtue of having (or having had) a parent or sibling with the illness; Who had never been tested for the HD gene, and who did not desire to be tested for the HD gene during the duration of the study; and Who had never been diagnosed with HD. How is confidentiality maintained in PHAROS? Confidentiality is a central concern in the PHAROS study, and we have designed the study to protect the confidentiality of research subjects to the fullest extent possible. Names do not appear on the study forms - instead, a code number is assigned to identify research participants. A bar code (different from the subject code) is used to identify blood samples and any DNA results. As mentioned previously, DNA results will not be provided to research participants or any of the physicians or staff at the site where the study is being conducted - instead, data will be transformed and sealed in a coded fashion (which does not identify individuals by name) and sent to a central secure location at the University of Rochester. Could eligible persons participate if they did not want to learn their DNA results or whether they might be developing signs of HD? Yes. Participants will never learn their DNA results through the PHAROS study, even when the study is complete. Participants can, if they later choose, arrange for DNA testing at an independent laboratory outside of the study. Research participants will also not be told if they are developing signs of HD. (If participants decide they do want to know this information, they will need to arrange for a clinical evaluation outside of the scheduled PHAROS study visits.) Are there any risks to PHAROS participants? Some psychological stress may occur from participation in PHAROS due to concern about showing signs of HD or carrying the HD gene. There are also some minor risks when blood is drawn for the blood sample. These risks are further explained in the consent form, and the research investigator or coordinator can also answer any questions. What is the benefit of participating in PHAROS? There is no direct health benefit from participating in PHAROS. However, participation may help to provide information useful for a better understanding of the onset of illness in unaffected persons at risk for HD. How do I find out more information about the PHAROS Study? As of January 2004, PHAROS completed enrollment. However, if you are interested in learning more about this study, please read our published articles. For a participating site near you, please go to our PHAROS Participating Site List. For access to the PHAROS Participant Newsletters, please see PHAROS Newsletter Volume Listing below. PHAROS UPDATE - July 28, 2006 At Risk for Huntington Disease. The PHAROS (Prospective Huntington At Risk Observational Study) Cohort Enrolled The Huntington Study Group PHAROS Investigators The Huntington Study Group (HSG) PHAROS (Prospective Huntington At Risk Observational Study) investigators reported on the baseline characteristics of this remarkable cohort in the July 2006 issue of the Archives of Neurology (63:991-998). PHAROS is a multi-site, multi-year project that aims to gain knowledge about the early signs of Huntington disease (HD) onset and their relationship to environmental and genetic factors. PHAROS is also examining the psychosocial, ethical and practical issues involved in long-term study of adults at 50:50 risk to develop HD who have chosen not to undergo predictive genetic testing to learn of their gene-carrier status. The PHAROS cohort of 1001 adults at risk for HD is mostly women (65%), well educated, and gainfully employed. At the start of the study, enrolled research participants were characterized as highly functional with minimal, if any, impairment of movement or intellect. Participants have enrolled in PHAROS mindful that the knowledge from this long-term study will help shape the efficient design and appropriate conduct of future clinical trials aimed at delaying the onset of illness in gene carriers who have not yet developed signs of HD. The knowledge from PHAROS will also inform us about how persons at high risk to develop a disabling genetic disease deal with lingering uncertainties about their future health and complex choices about their participation in research. Related Documents: PHAROS Participant Thank you Letter August 2009 PHAROS Participating Site List PHAROS Newsletter Volume 1, 2004 PHAROS Newsletter Volume 2, January 2005 PHAROS Newsletter Volume 3, July 2005 PHAROS Newsletter Volume 4, March 2006 PHAROS Newsletter Volume 5, August 2006 PHAROS Newsletter Volume 6, March 2007 PHAROS Newsletter Volume 7, December 2007 PHAROS Newsletter Volume 8, June 2008 Huntington Project Research Spotlight report 'Review of Published Article-At Risk for Huntington Disease: The PHAROS Cohort Enrolled-', July 2007 Related Links: ClinicalTrials.gov July 2006 issue of the Archives of Neurology (63:991-998) last updated: August 2009	Thank you PHAROS study participants! The HSG and the PHAROS Steering Committee would like to thank all of the PHAROS study participants. (read more...)