Frequently
Asked Questions
What are clinical trials?
Clinical trials are a type of research designed to answer specific medical questions such as, is this treatment safe for patients or how well does the treatment work? The research follows a very specific process and involves testing the new drugs, medicines, or devices on real people.
Why should I consider enrolling in a clinical trial?
Participants in clinical trials help researchers find out if new medicines or devices are safe and truly help the patient. They provide new information about a disease or process that can help current and future patients live a fuller life. Being in a trial might also give you access to new treatments before they are approved and available to others.
How do I get notifications about new clinical trials?
The website, www.ClinicalTrials.gov, provides a list of clinical trials in the US. You can select a specific disorder on their site and sign up for notification of new trials. Parents or caregivers of an individual diagnosed with Angelman syndrome can join the Global Angelman Syndrome Registry or the LADDER database and sign up for clinical trial notifications. This site is also a great place to find studies looking for participants.
Who is eligible to participate in a clinical trial?
Each clinical trial wants to answer a specific question. That’s why researchers provide a list of eligibility requirements and people who meet the requirements may be enrolled in the trial. Each study is different so you may qualify for one trial but not the other.
If a person is eligible for a trial, how do they enroll?
If someone meets the eligibility criteria for a clinical trial, they must use the information provided in the trial listing to contact the study organizers. After reviewing information, the trial organizers may want to set up a meeting or have you complete eligibility tests. If the team thinks you meet the requirements, they will help you enroll in the study.
What is an informed consent document?
Once you are approved for a clinical trial but before any treatment begins, the study organizers will give you an informed consent document. This document provides all details of the trial including:
How the study will be conducted
Specific participant requirements
Risks and benefits of participation
Study duration
Cost to you, if any
Payment to you, if any
Names and phone numbers of key contacts
After reviewing this information and asking any questions, you sign the consent form if you wish to proceed with the study. If you decide not to continue with the trial, return the document to the organizers without signing it.
What happens during a clinical trial?
Every trial follows a defined protocol carefully designed to answer a medical question. Some studies involve observing behavior and taking surveys, while others require specimen collection, medical tests, and treatments. (Details are in the informed consent document.) All clinical trials gather the participants’ personal information and monitor them closely throughout the entire study period.
Do the trial medicines always work?
Less than 14 percent of all drugs in clinical trials receive approval from the FDA*. That means the majority of investigational medications never make it to the consumer. But that’s the point. When a new drug or medical device is studied for the first time in people, scientists don’t know exactly how the patients will react. They depend on study participants to help identify areas of success and improvement. (*January 2018 study from MIT Sloan School of Management in Biostatistics)
How long will the trial last?
Each clinical trial asks a question. Some questions will be answered more quickly than others. That’s why the length of each trial is different. Details for each particular study can be found in the informed consent document.
Can you be in two clinical trials at the same time?
There are no laws against participating in more than one clinical trial at the same time. However, you should check with the PI of the current clinical trial you are participating in (or in each trial you are interested in) to see if that is an exclusion criteria.
Where is the trial? Do you have to travel?
Most clinical trials take place where people already go for medical care – a doctor’s office or clinic. These locations can be near you or require travel. The details for each study can be found in the informed consent document.
Will the trial sponsors pay for my travel expenses?
Not all studies pay for travel to and from the trial site. When travel is included, payment takes many forms including reimbursement for gas or taxi services. Some communities use Uber, Lyft, and similar companies to transport participants to the study location. Details related to travel costs will be in the informed consent document and you can ask about compensation for trial costs at any time. Please note that the IRS requires study payments of $600 or more to be reported on tax returns.
Will the trial sponsors pay me to be in a clinical trial?
No rule or regulation requires sponsors to pay study participants; however, many offer compensation of some kind. Money, travel reimbursement, free health care, free screening exams and other tests are the most common forms of compensation. Details related to payment are in the informed consent document. Please note that the IRS requires study payments of $600 or more to be reported on tax returns.
What happens once the trial is over?
Data collected from the trial determines if the drug, device, or treatment was successful. Studies that show success can move on to the next phase of clinical trials. Generally, this involves enrolling more people in a new trial. For participants, the researchers will contact you and share conclusions from your trial. For studies interested in long-term effects, surveys and periodic health exams may be requested.
Can you continue getting a trial drug after the study is over?
While it’s not a guarantee, there are instances where study participants continue to receive the study medication in a follow up study, especially if it’s a medication for a rare disease or the participant has experienced significant improvement and termination of the drug would cause an overall decline in the person’s health.
Can you be in a clinical trial if you live outside the United States?
Each study provides very specific criteria a person must meet in order to participate in a clinical trial. Living in the United States may be one of those eligibility requirements. The trial listing/description and the informed consent document will tell you if geography is a determining factor of participation
What if my loved one doesn’t qualify for a clinical trial?
If your loved one does not qualify for a study right now, be patient. There are Angelman syndrome research studies happening at universities and medical centers all over the world.
Can you share information about your experience on social media or publicly?
No. Please keep your experience with the study confidential throughout the life of the clinical trial. Participants and their immediate family members should not share information about clinical trials by phone, in conversation, or on social media platforms. Most trial sponsors will strongly discourage anyone communicating either positive or negative experiences. This will help avoid any type of study bias. They want to do everything possible to have a successful study and potentially bring products to market.