Clinical Trial FAQs
What are the possible benefits of being in a clinical trial?
- You may get access to a new therapy, sometimes years before it is widely available.
- You will be closely monitored for side effects
- You will be helping other patients and families living with your rare disease
What are the possible risks?
There is always some risk involved in clinical trial participation. Researchers try to balance the possible benefits of the trial with the risks that participation poses. They also conduct studies to see if the treatment is likely to be safe and effective before testing it on people. It’s still possible that:
- You could experience side effects
- The treatment might not work for you, even if it works for others
- You might have a reaction that means you have to stop treatment, even if it was previously working
- Participating in the clinical trial may take more time than the standard of care and may involve travel
- You could develop a reaction to the vector that delivers the gene therapy
How will my rights and safety be protected?
In the US, every clinical trial must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits.
IRBs usually include doctors, nurses, statisticians, community advocates, and others who make sure that your rights are protected. The IRB approves a plan, called the protocol, which researchers agree to follow. You will be informed about the protocol before you enroll in the trial and this information will be available to you throughout the trial.
What is informed consent?
Informed consent is the process of agreeing to be in the clinical trial. The research team will explain the trial’s purpose, how long it will take, what will happen, all potential risks and benefits, and information on the privacy of your medical records. If you want to participate, you will sign a consent form. Informed consent is not a one-time event. It continues throughout the trial. You have the opportunity to ask about the trial as it progresses.
Can I quit a clinical trial?
Yes, you can leave at any time, for any reason.
How do I know if I'm eligible for the clinical trial?
Every clinical trial has guidelines that determine who can participate. These “eligibility criteria” can include age, gender, stage of the disease, genetic mutation, previous treatment, and other medical conditions. Researchers are careful about screening participants and including only those who fit the criteria. This helps keep you safe and ensures that researchers get the information they need.
How will researchers know if the gene therapy is working?
In every study there are at least two big sets of data:
- The first describes what is happening to the whole person. Is the patient experiencing symptoms? Has the patient avoided side effects?
- The second data set includes the lab tests (biological assays) that are used to measure effectiveness of the cell therapy, gene therapy, drug, or treatment.
How you feel is important, but it may not be enough to prove that the gene therapy is effective. The FDA wants to see consistency in the effect of the drug as measured by lab tests before they can move to the next phase of the study, and have more people participate.
What if I participate in the clinical trial and the treatment is not effective?
If the effectiveness of the gene therapy isn’t verified by lab tests, it is likely that you will be returned to the standard of care, even if you are experiencing improved health.
If gene therapy does improve the way my body functions, how long will the effect last?
Gene therapies affect the way your body functions by introducing normal copies of genes that will produce the right protein in the right amount. If they continue to produce this needed protein over time, and your medical condition remains improved the effect is said to be “durable.”
One of the goals of clinical trials is to measure durability. If researchers find that the ability of the gene to produce the protein decreases over time, they will try to understand what is needed to produce a lasting response. Sometimes the trial will stop, or not advance to the next stage if the response is not long lasting.
I feel good. Am I done now?
Even if you or your child starts to feel better, lab tests and monitoring continue so that researchers can determine whether or not the effect of the treatment is durable.
Will I need more than one treatment?
Many gene therapies are designed to be one-time treatments. But for some diseases, re-dosing may be required. So, another question that the clinical trial is designed to answer is whether or not redosing will be necessary to maintain the effect. Scientists will not know whether or not redosing will be required until the trial is complete.
What if I develop a reaction to the treatment?
There is a risk of side effects and adverse reactions with any treatment. Because of this, doctors and researchers carefully monitor study participants for any negative health impact and try to determine whether or not the adverse reaction was caused by the treatment.
Most current gene therapies have 2 critical elements, the gene to be introduced, and the vector (gene delivery vehicle) that helps the new gene get into the cell nucleus. You may have an immune response to the vector or the new gene. The response might make the gene therapy ineffective, or the gene therapy might trigger a bad reaction. If this happens, you may be taken out of the study.
If this gene therapy doesn't work, can I try another?
It depends. There are a number of factors that will determine whether or not you’re eligible for future trials.
With rare diseases, your doctor is unlikely to recommend your participation unless she feels that it offers the right approach for you. Nevertheless, there is a chance that your participation in this trial may prevent you from participating in a future trial (e.g. if you build up resistance to the vector used to carry the DNA.)
How long will I need to participate in the study?
Gene and cell therapy studies are intended for people who are willing to participate for the rest of their lives. Researchers can be sure that the effect of the gene therapy is durable and that other problems don’t arise down the line.
What is involved in participating long-term?
You may be required to schedule doctor visits and blood tests that will provide ongoing data for the study. This is true even if you feel better. You might even have emergency visits that you didn’t anticipate. People who participate in clinical studies give back to the community through their willingness to commit to a protocol for their whole lives.
Do I have to pay for my treatment in the clinical trial?
No, but you may have to cover some costs. The treatment and all associated visits with healthcare professionals are ordinarily paid for by the company that is sponsoring the clinical trial. Health problems connected to trial participation, such as an adverse event, are usually covered as well.
You or your insurance company will be responsible for the cost of any treatment, procedures, or tests that are part of your routine care, whether you are in a clinical trial or not.
Do I have to pay for travel to the trial site?
Because you will have to be closely monitored by the study coordinator and the team of healthcare professionals working on the trial, you will either have to live close to the trial site or plan for regular travel. Some clinical trials will reimburse you for needed travel and lodging, but many will not.