Nav: Home

When designing clinical trials for huntington's disease, first ask the experts

April 23, 2019

Amsterdam, NL, April 23, 2019 - Progress in understanding the genetic mutation responsible for Huntington's disease (HD) and at least some molecular underpinnings of the disease has resulted in a new era of clinical testing of potential treatments. How best to design clinical trials in which HD patients are willing to participate and comply is a question faced by researchers. For that reason, investigators in the Perelman School of Medicine at the University of Pennsylvania (Penn) surveyed HD patients at different stages of the disease about their attitudes and treatment goals. The results, published in the Journal of Huntington's Disease, should be useful for designing future clinical trials of gene therapies for HD and other genetic disorders.

"Clinical trials in HD are creating a sense of optimism and hope within the HD community, with the majority of respondents [to our survey] reporting willingness to participate in trials. As new trials are developed and implemented, it is important to take the opinions and attitudes of the patient population into account," explained Tanya M. Bardakjian, MS, CGC, senior genetic counselor in the department of Neurology at Penn.

Treatments now being tested can be nontraditional, going beyond taking a daily pill. Some require repeated intravenous infusions, lumbar punctures, or brain surgery; some may be administered only at sites far from where the patient lives; and others may result in significant side effects. "In order to optimize feasibility, it is important to first understand how potential study participants feel about these new trial designs, with interventions that might involve a higher potential risk but also higher potential reward," noted Ms. Bardakjian. The authors also wanted to determine whether responses differed if the respondent was a potential disease carrier, an asymptomatic genetic carrier, or showed HD symptoms.

Eighty-seven respondents completed an anonymous survey, including 36 patients diagnosed with HD, 18 pre-manifesting mutation carriers, and 33 asymptomatic participants at risk. The questionnaire included multiple-choice questions concerning hypothetical scenarios. For example, one question asked, "How likely are you to participate in gene therapy trials if treatment was an IV infusion in the arm requiring a clinic visit once a month?" Answers could range from not likely to very likely.

Another question assessed the respondent's willingness to participate in a clinical trial based on personal goals of therapy such as seeking a cure, slowing disease progress, or helping future generations. They were also asked about how they perceived the investigators' motivation to conduct trials, including whether they thought investigators were seeking financial gain or looking to advance their careers. Results were broken down according to the three disease-status groups described above. The questionnaire also collected demographic, clinical, and genetic background data.

The responses indicated that the majority of participants were very likely or likely to participate in clinical trials, regardless of study design or goals of therapy. However, individuals who know they carry the mutation and therefore will develop HD in the future but have no symptoms yet, had the most favorable views. Additionally, more invasive procedures and trials that include administration of a placebo were viewed less favorably across all groups. Ms. Bardakjian suggests that one way to counteract the negative effect of including a placebo group within a trial is to include the option of receiving active treatment once the placebo period ends.

"We believe that a patient-centered trial design, through the inclusion of appropriate education and communication to ensure participants and advocates are informed and engaged, is likely to have a positive impact on recruitment," commented Ms. Bardakjian.
-end-
The work was supported by a grant from the Personalized Medicine Special Interest group of the National Society of Genetic Counselors.

IOS Press

Related Clinical Trials Articles:

Review evaluates how AI could boost the success of clinical trials
In a review publishing July 17, 2019 in the journal Trends in Pharmacological Sciences, researchers examined how artificial intelligence (AI) could affect drug development in the coming decade.
Kidney patients are neglected in clinical trials
The exclusion of patients with kidney diseases from clinical trials remains an unsolved problem that hinders optimal care of these patients.
Clinical trials beginning for possible preeclampsia treatment
For over 20 years, a team of researchers at Lund University has worked on developing a drug against preeclampsia -- a serious disorder which annually affects around 9 million pregnant women worldwide and is one of the main causes of death in both mothers and unborn babies.
Underenrollment in clinical trials: Patients not the problem
The authors of the study published this month in the Journal of Clinical Oncology investigated why many cancer clinical trials fail to enroll enough patients.
When designing clinical trials for huntington's disease, first ask the experts
Progress in understanding the genetic mutation responsible for Huntington's disease (HD) and at least some molecular underpinnings of the disease has resulted in a new era of clinical testing of potential treatments.
New ALS therapy in clinical trials
New research led by Washington University School of Medicine in St.
Telemedicine helps improve participation in clinical trials
Videos and creative uses of other visuals provide a novel way to obtain informed consent during clinical trials to improve participants' understanding and retention of trial information, according to a study by Nemours Children's Health System presented at the American Thoracic Society (ATS) Annual Conference.
Not enough women included in some heart disease clinical trials
Women are underrepresented in clinical trials for heart failure, coronary artery disease and acute coronary syndrome but proportionately or overrepresented in trials for hypertension, atrial fibrillation and pulmonary arterial hypertension, when compared to incidence or prevalence of women within each disease population, according to a study in the Journal of the American College of Cardiology.
BU: Obese patients underrepresented in cancer clinical trials
A new review by Boston University School of Public Health researchers found that less than one-fifth of participants in cancer-related clinical trials are obese.
Are women really under-represented in clinical trials?
Several studies have reported a lack of gender diversity in clinical trials, with trials including mostly adult males; however, a recent review of publicly available registration data of clinical trials at the US Food and Drug Administration for the most frequently prescribed drug classes found no evidence of any systemic significant under-representation of women.
More Clinical Trials News and Clinical Trials Current Events

Top Science Podcasts

We have hand picked the top science podcasts of 2019.
Now Playing: TED Radio Hour

Risk
Why do we revere risk-takers, even when their actions terrify us? Why are some better at taking risks than others? This hour, TED speakers explore the alluring, dangerous, and calculated sides of risk. Guests include professional rock climber Alex Honnold, economist Mariana Mazzucato, psychology researcher Kashfia Rahman, structural engineer and bridge designer Ian Firth, and risk intelligence expert Dylan Evans.
Now Playing: Science for the People

#541 Wayfinding
These days when we want to know where we are or how to get where we want to go, most of us will pull out a smart phone with a built-in GPS and map app. Some of us old timers might still use an old school paper map from time to time. But we didn't always used to lean so heavily on maps and technology, and in some remote places of the world some people still navigate and wayfind their way without the aid of these tools... and in some cases do better without them. This week, host Rachelle Saunders...
Now Playing: Radiolab

Dolly Parton's America: Neon Moss
Today on Radiolab, we're bringing you the fourth episode of Jad's special series, Dolly Parton's America. In this episode, Jad goes back up the mountain to visit Dolly's actual Tennessee mountain home, where she tells stories about her first trips out of the holler. Back on the mountaintop, standing under the rain by the Little Pigeon River, the trip triggers memories of Jad's first visit to his father's childhood home, and opens the gateway to dizzying stories of music and migration. Support Radiolab today at Radiolab.org/donate.