FREQUENTLY ASKED QUESTIONS
Here is a list of the most frequently asked questions we have received, if you have any please feel free to ask them below.
Clinical trials (also referred to as clinical studies or more broadly as clinical research) are research studies involving human volunteers. In order to test a drug in humans, European Medicine`s Agency (EMA) has to approve the trial. This is to make sure all trials in humans are done in a scientifically sound, ethical and safe way.
EMA and 50 regulatory authorities from the 31 EEA countries (28 EU Member States plus Iceland, Liechtenstein and Norway) and the European Commission require that a potential therapy has to be tested extensively in a large group of human volunteers before it can receive approval to be manufactured and made available to patients.
All trials and studies on HDTrialFinder relate to Huntington’s Disease (HD) and have been approved by the regulatory authorities for human testing. These trials assess the safety and effectiveness of new ways to diagnose, prevent, or treat HD; they also provide insight into the disease process, and how it might be treated. Clinical trials are a vital part of the scientific research process and are essential to developing better treatments for people affected by Huntington´s disease.
There are two main types of clinical trials:
1) Observational clinical trials: do not test drugs or treatments. Researchers observe participants by monitoring their health over a period of time. These studies provide researchers with data that advances our understanding of Huntington’s and how to treat the disease.
2) Interventional clinical trials: test the safety and effectiveness of a candidate drug, therapy or experimental treatment
Clinical trials and studies play a critical role in the development of new and better therapies. Yet under-enrollment (not enough participants) in trials is one of the greatest challenges clinical researchers face. Under-enrollment in trials slows research progress and deters potential funders from investing in research — and we all pay the price in terms of higher costs and longer time horizons to get to therapeutic breakthroughs. Across all diseases, 85 per cent of clinical trials finish late due to difficulties enrolling participants and nearly one-third of trials fail to recruit a single subject and cannot even begin.
In spite of this low participation rate, we know that the Huntington community has a significant interest in stepping up to participate in research. Patients not knowing about specific trial opportunities in their area, that need someone like them, is one the greatest barrier to trial participation.
HDTrialFinder is one solution to help patients and their loved ones act on the intention to get involved in research by making it easier to find trials near them— and letting volunteers know when new trials start that could be a good match.
Yes. Although ideally, all study participants should remain enrolled until the study’s completion, circumstances, disease progression and other factors may contribute to a volunteer’s decision to leave a trial. This is permissible at any time. To withdraw from a study, a volunteer should immediately inform the trial coordinator of this decision.
Due to ethical and practical considerations, every trial has its own policy regarding volunteer compensation. Some trials may reimburse volunteers for expenses related to their participation or even offer some compensation to volunteers for participating. Some trials that require significant travel or an overnight stay may cover the associated expenses. A trial team member can provide more details on whether compensation is available as part of trial participation.
Every trial follows an extensive and carefully monitored protocol subject to EMA regulations, many of which focus on the safety of the participants in a trial. The protocol is a detailed plan that describes the study process. In addition to the protocol, each study has a consent form (also sometimes referred to as informed consent or consent to participate) that volunteers must read and sign before joining the study. The consent form will explain the rights and responsibilities of a volunteer and what to expect during the study. Study protocols and consent forms must be reviewed and approved by a regulatory body before any volunteers can be approached about or recruited into a study to ensure that the study is safe, ethical and appropriate.
Trial team members will discuss the consent form and any aspects of the protocol in detail during a trial screening visit. Volunteers can also request a copy of the consent form to review it in advance of this in-person visit.
Once a clinical trial is over, the trial team assesses the data, reviewing the key findings, publishes or presents any novel findings and determines the appropriate next steps for future testing, as appropriate. They may continue to evaluate the treatment in the next trial or discontinue research because the treatment has not been shown to be safe or effective. In some cases, if the treatment continues to the next phase, trial participants will be given the option of participating in that phase of the study as well.
Volunteers can search the Web or follow up with the trial team to inquire about study outcomes.