1800 244 735

Helpline (02) 9874 9777

Improving Huntington's disease clinical trial recruitment through patient and family education

Trials of new treatments for Huntington’s disease are happening, but proving whether a drug works requires lots of suitable volunteers. Surprisingly, finding enough volunteers is often difficult. Now a group of HD professionals has shown that education and outreach to patients in the community works well for boosting recruitment.

Progress towards effective treatments for Huntington’s disease feels agonizingly slow for the people who need them most. But clinical trials to test new treatments for Huntington’s Disease are happening. Before a drug can be approved for use in HD, it must be tested in symptomatic patients and shown to have a benefit. The improvement must be robust enough that it’s clear that the drug worked. That can require a large number of subjects, because the more people you test a drug in, the more likely you are to have conclusive results.

It may surprise some people in the HD community to hear it, but finding enough volunteers for HD drug trials isn’t easy. Motivated by this concern, a group of HD professionals, lead by Dr LaVonne Goodman, set out to find ways to improve recruitment of volunteers to participate in HD clinical trials.

The Trial

The trial that Goodman and her team set out to help fill was the recently completed “HART” study of Huntexil for movement symptoms in Huntington’s disease.

The scientists who planned the HART trial calculated in advance that 220 volunteers were needed to test the drug conclusively. They would be recruited from 28 clinics in the USA and Canada. Each volunteer would remain in the trial for three months.

Recruitment

Recruitment for HART in the USA was slow. It took nearly 2 years (from October 2008 to August 2010) to find 220 volunteer patients. For comparison, a trial in Europe with the same drug, called the ‘MermaiHD’ trial, recruited 420 HD patients in less than a year.

Improving that slow rate of recruitment for clinical trials would shorten the delay in obtaining trial results. To help with this, Goodman and her colleagues set out to understand what factors help HD families get involved in trials.

In an earlier study, with collaborators from the Huntington’s Disease Society of America and CHDI Inc., Goodman had set out to discover how much patients and their families understand about HD. By surveying patients and family members at local chapter meetings, she showed that the desire for information about trials, and the motivation to participate in them, was very high. What was missing was information for families about available trials. Goodman concluded that “education in support groups is a mechanism that can increase clinical trial participation”.

An opportunity to test these ideas was presented when the Evergreen Neuroscience Institute became a new Huntington Study Group site involved in the HART trial. With no regular HD patient base of its own, the site needed to recruit in new ways. Community advocates, educated about trials in general and Huntexil in particular, met with small groups of HD patients and families. In three sessions they had group discussions about the risks and benefits of this research. At the end of these educational sessions, printed information was available to interested parties.

In addition to this in-person work, the HDSA sent pamphlets to members of its mailing list. People who had offered to participate in trials were also alerted via a website, HDTrials.org, that exists to facilitate this kind of communication. Finally, HDSA websites were updated with information about the trial.

Results

So which of these techniques was most successful in terms of encouraging willing and able participants to enroll in the trial? The Evergreen Neuroscience Institute saw seventeen HD patient volunteers, 13 of whom completed the HART trial. This made Evergreen the third largest of the twenty-eight sites in the HART trial – pretty impressive, considering that they had no regular HD patients to begin with. They were also the fastest site in the study in terms of recruitment rate. Clearly, Evergreen Neuroscience Institute was doing something right.

Where did their seventeen volunteers hear about the study? More than half were recruited by the in-person local advocacy work. Meeting families and talking to them about clinical research was Evergreen’s most effective way of recruiting patient volunteers. The other sites involved in the HART study had a different experience: most of the other volunteers in the HART study came through direct referral by professionals working at the site.

Take home message

It seems that, in this case, working in person with families was the best way to enlist volunteers for clinical trials. The entire HD community benefits from clinical trials, because whether they succeed or fail, they bring us closer to the day when treatments are available for everyone. In future, there will be even more trials, many running at the same time, and getting enough patients involved is a challenge for the entire global HD community. Goodman and her team have shown one way to speed recruitment of volunteers to run successful trials.

Latest Research Articles

Focusing in on fibrils; scientists give us a glimpse of huntingtin protein clumps

Published date: 8 September, 2022

A group of scientists from the EPFL in Lausanne, Switzerland have published a paper in the Journal of the American Chemical Society, describing clumps made up of a fragment of the huntingtin protein. A word that’s commonly used to describe these is “aggregates.” Using very powerful microscopes, the team was able to zoom in and ... Read more

Hereditary Disease Foundation (HDF) conference 2022 – Day 4

Published date: 2 September, 2022

DNA repair and CAG repeat instability The effect of HTT lowering on CAG repeat expansions Welcome to last day of the @hdfcures conference! We’ll only be sharing a few talks from today’s sessions, which focus on DNA repair. The first is from HDBuzz’s very own Jeff Carroll! Jeff will be sharing his work on HTT ... Read more

Hereditary Disease Foundation (HDF) conference 2022 – Day 3

Published date: 1 September, 2022

Pre-clinical work moving toward trials New tools to lower HTT showing promise in animal models Welcome back! The first talk we will be tweeting about today is from Anastasia Khvorova, who will be telling us about her teams work on lowering of Huntingtin using technology called RNAi. One of the problems in studying drug delivery ... Read more

Hereditary Disease Foundation (HDF) conference 2022 – Day 2

Published date: 31 August, 2022

We’re back for day 2 at @hdfcures! This morning’s talks will be focused on clinical trial planning and therapeutic updates from clinical studies. The sheer number of talks related to human trials compared to previous years is so encouraging! Updating metrics for clinical trials A better system for disease categorization The first talk of this ... Read more

Hereditary Disease Foundation (HDF) conference 2022 – Day 1

Published date: 31 August, 2022

Hello and welcome from the HDBuzz team who are currently at the Hereditary Disease Foundation (@hdfcures) 2022 Milton Wexler Biennial Symposium in Boston! It’s the dawn of an exciting new era for HDBuzz. Due to our new partnership with @hdfcures, we are now able to live tweet many of the talks from this meeting which ... Read more

Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials

Published date: 29 August, 2022

Last month, we relayed positive news from uniQure’s trial testing AMT-130, a gene therapy delivered via brain surgery to lower huntingtin (HTT). Data released by uniQure in June suggested AMT-130 was safe and well tolerated in the small group of people that were treated with a low dose of the drug. Now we’re back to ... Read more