About This Study
Enroll-HD is a worldwide observational study for Huntington’s disease families. It will monitor how the disease appears and changes over time in different people, and is open to people who either have HD or are at-risk.
Enroll-HD is or will soon be up and running for HD families in North America, Europe, Latin America, Australia, New Zealand and some countries in Asia. It will eventually include more than 20,000 people. Monitoring people over time in a real-world setting contributes to scientific knowledge. The study is designed to accelerate the discovery and development of new therapeutics for HD.
We are building this infrastructure for all HD researchers to use with three main goals:
- First, to better understand Huntington’s disease as it happens in people , to give us insights into developing new drugs
- Second, to improve the design of clinical trials to give us clear answers more quickly. We want better, smarter, faster clinical trials so that we can move as quickly as possible to find the treatments that work
- Third, to improve clinical care for HD patients by identifying the best clinical practices across all Enroll-HD sites around the world and then working to ensure that all families get that standard of care
HD symptoms often change slowly, so observational studies are essential for documenting how things usually play out over the course of the disease. Knowing how HD typically unfolds will make it much easier to evaluate more quickly whether a new experimental drug is working to slow down or stop those typical changes. Enroll-HD is a prospective observational study, meaning it tracks people over time, rather than asking them to recall how their symptoms have changed.
Enroll-HD will help accelerate research by:
- Collecting standardized data, in the same way and using the same methods, from tens of thousands of people around the world. More people undergoing the exact same evaluations means the study is more likely to uncover new things about HD.
- Making data available to any scientist with a legitimate research project who wants to better understand HD. We want as many scientists as possible all around the world working on HD.
- Developing a comprehensive database of people who may be interested in volunteering for future studies, including drug trials, with all the necessary information about them already in place.
Enroll-HD is a “platform”
The idea is that this one study will make all other HD research easier, speeding up the process of developing new drugs and other treatments that really work. Rather than being restricted to a select group of researchers, it will be a resource for all HD science, making it faster and more efficient for other researchers to conduct their projects.
This is why it’s called a platform, it supports other work. Think of it this way; a smartphone is a platform that supports apps that do lots of different things. Enroll-HD is the platform that will support lots of different research studies and clinical trials.
Essentially, Enroll-HD lays out a “welcome mat” for researchers and pharmaceutical companies to study HD, supplying many of the crucial ingredients for a clinical trial — this includes a global network of research sites, a carefully maintained database that tracks people’s health over time, and an up-to-date privacy-protected list of people who might want to volunteer for a new study.
For more about what it means to call this study a “clinical research platform” and how it will help unify the HD research community, read the article Building on Enroll-HD, originally published in the Enroll! newsletter.