About this guide
This resource, “Guide for considering participation in an interventional clinical trial,” is specific to interventional studies or interventional clinical trials. An interventional trial is a research study that investigates a drug or other treatment for its safety and efficacy in a human population.
For clarity, we will refer to the person taking the drug or accepting the treatment as “the participant” whether this person is your loved one or yourself.
Purpose of this guide
The basis of voluntary participation in any trial of an investigational drug or treatment is informed consent. This means the participant has all the information they need to make a calculated decision that weighs the potential risks and benefits before consenting to participate in the trial. You will be asked to sign an Informed Consent Form, acknowledging that you understand the risks and benefits of participating in the trial before you are able to be enrolled in the study.
This guide is intended to help you ask the right questions to fully understand the potential risks, benefits, and costs of participating in the trial of a new, investigational drug or treatment.
The ARRE Foundation encourages you to work closely with a medical provider whom you trust and who is familiar with the participant’s condition. Your provider should help you find answers to the following questions, understand and evaluate the answers, and guide you through the trial process.
- Has a medical provider who you trust asked rigorous scientific and medical questions of the trial sponsor (the entity funding and running the trial) and of the clinical trial lead (the person in charge of administering the treatment to the trial participant)? These questions might include:
- Is there peer-reviewed scientific evidence that supports the proposed treatment approach?
- Does this evidence support the unique ASXL variant (gene mutation) the participant has?
- Do you understand the potential risks and benefits of this treatment, including what risks and benefits are unknown?
- Do you understand how this trial will be carried out and how it will impact your family, including how the treatment will be administered (put into your child), how often dosing of the treatment needs to occur, how long the trial period is, and what monitoring and follow-up is required?
- Do you understand who is financially responsible for the costs of administering the treatment and any follow-up care during the trial period?
- Do you understand what access you will have to the treatment after the trial if it is successful, including who is responsible for the ongoing costs of the treatment and administration of the treatment once the trial has ended?
- Do you understand how the trial is regulated, including who approves the trial to go forward based on the scientific evidence? All details of the study should be available on ClinicalTrials.gov.
- Do you know whom to inform if you wish to terminate participation in the trial for any reason? It should be explained to you that your access to healthcare during and after the trial will not be affected regardless if you complete the trial or withdraw early.
We’re here to help
If you are considering participation in an interventional trial and you or your doctor have additional questions, please contact the ARRE Foundation at info@arrefoundation.org.
The information presented here is for educational purposes only. This information is not medical advice and should not be treated as such. You should always talk to your or your family member’s health care professionals for diagnosis and treatment, including information regarding which drugs or treatment may be appropriate for you or your family member. We do not represent or warrant that any particular treatment is safe, appropriate or effective for you or your family member. In the event of a medical emergency, you should contact your doctor immediately or call your local emergency service.
