What is Enroll - HD?
Enroll-HD is a so-called "observational" clinical study: it consists of clinical data and biological samples collection, without administration of any experimental drug.
It is carried out in all five continents and is funded by the CHDI Foundation, which is based in the United States.
What is the goal of Enroll-HD?
The objective of the study is to understand the natural history of the HS, transforming a series of insights into numbers.
What does it consist of?
Those who agree to participate are required to undergo an annual visit, which consists of a medical and psychological monitoring, the administration of tests to assess behavior, movement, emotional status and quality of life and a blood sample, which is NOT a genetic test.
Where do my data and blood end up?
All information collected flows into a secure computer system, which is located in the UK. It is the same system used by banks and hospitals. Blood samples, instead, are sent to a central biobank, called BIOREP, located in Milan. Researchers all over the world can request access to these data and samples, but they will never read any names, just numerical codes.
How long does the visit take?
The first visit (called baseline visit) is the longest and lasts about 2 hours - 2 hours and a half. Subsequent visits (called follow-up visits) last from 45 minutes to 1 hour and must be made between 9 and 12 months after the previous visit. To allow researchers to assess any changes that may occur over time, it is essential not only to enter the study, but to remain enrolled in it over the years. To this end, an efficient planning of the annual visits as well as the collaboration of participants and any accompanying persons (caregivers) are essential.
How long does the study last?
The study does not have a completion date. In order to be effective, it is recommended that it should last many years, or at least as long as possible. Those who wish to leave the study can do so, but this choice will take away useful insights for the progress of research.
How is Enroll-HD useful for research?
Enroll-HD can be defined as a real research platform: it is designed to make all other studies on Huntington's disease faster and more efficient, for the following reasons: - data are collected in a standardized way and with the same methodology throughout the world, so as to provide an extremely accurate representation of the disease; - all interested researchers can ask to access the data they intend to study to verify hypotheses; - Enroll is a huge database of individuals potentially eligible to participate in pharmacological clinical trials
Are Enroll-HD and clinical trials connected?
When someone decides to participate in Enroll-HD, he or she is asked to indicate on the informed consent form whether they are available to participate in other clinical and pharmacological studies on Huntington's disease. In this case, should the possibility of an experimental drug therapy arise, the neurologist/researcher at the Huntington Center in charge will notify this opportunity. Accepting or refusing to take part in therapeutic trials remains a free choice. Obviously, this opportunity concerns only those who carry the genetic mutation. Those who do not know their status - because they have not done and/or do not intend to do the genetic test - cannot participate in therapeutic trials, unless these are specifically designed for people 'at risk'.
Who is eligible to participate?
It is not necessary to be a gene carrier to take part in this research program. The study, in fact, is open also to people at-risk who are not willing or could not undergo a genetic test, to unrelated partners and even to people under 18 years of age, but only if they manifest clinical symptoms.
Why should I participate?
By participating in Enroll, you provide researchers with invaluable information about the evolution of the disease, whether you are affected or not. Huntington's disease is a rare disease and to fully understand its manifestations and progression it is fundamental to know the numbers, in order to study it on a large scale. Numbers will make the difference. We have to engage a lot of people. The privacy of participants is protected in a very strict way: a highly complicated computer system transforms participants’ names into numerical codes, making it impossible to trace their identity.
What if I have an economic difficulty in moving?
A flat-rate reimbursement of participation costs is envisaged: from a minimum of 20 to a maximum of 60 € per participant, depending on the distance in kilometers to reach the Huntington Center. Refunds are given in cash on the day of the visit.
I want to participate, what should I do?
To participate, simply call our toll-free number 800.388.330 or ask directly to the LIRH staff, when you come to the visit or accompany a family member on a visit.