Pediatric Clinical Trials & Experimental Medication
Clinical trials offer the latest, most promising new therapies. At Children’s Health℠, we offer more clinical trials than almost any other center in our region – for everything from cancer to epilepsy.
Our UT Southwestern physicians and researchers are constantly investigating new treatments and launching new clinical trials. This gives our patients access to every possible treatment option. And it helps us continue improving and redefining pediatric health care.
What are Pediatric Clinical Trials & Experimental Medications?
Clinical trials are research studies that evaluate if a new treatment is safe and effective for people. Volunteers of all ages participate in clinical trials (also known as medical research studies).
Experimental medications are either brand new medicines, or existing medicines that are being used in a new or different way.
Not every child does well with their current treatment plan or medication. And, unfortunately, there aren’t effective therapies for every condition. Clinical trials and experimental medications are ways for patients to try new therapies that can potentially help the child. Participation in clinical trials can also help others with the same condition by contributing to science and helping researchers find better treatments in the future.
Families from across the U.S. come to Children’s Health to participate in our clinical trials.
Learn more about the types of Pediatric Clinical Trials & Experimental Medication we offer
Generally, we offer two types of clinical trials:
- Clinical trials of medicines that have been approved for adults but have not yet been studied for children. Our researchers use clinical trials to offer this type of medicine to children. We gather accurate, reliable data because we need to know if the treatment works and what is the medication dosage that works best for children. In a trial like this, your child may get the medication being studied or they may be part of the control group, which means they receive the current standard of care treatment.
- Trials of medicines that aren’t yet FDA approved. This is traditionally what people think of as experimental medication. The medicine may or may not turn out to be effective. There are several phases of these trials. Initially, Phase 1 and Phase 2 trials aim to determine if the medication is safe for children. Later, Phase 3 and Phase 4 trials look more closely at whether that medicine is effective and should be approved for broader use.
What are the benefits of Pediatric Clinical Trials & Experimental Medication?
A clinical trial could benefit your family because it offers an opportunity to try a new medicine or one that’s used in a new way. Many families also join clinical trials because they know that the results could help children in the future.
We can’t guarantee that the experimental medication will help your child. But we can promise you that when your child is in a trial, it gives doctors and researchers information to help other children. A lot of parents and children like knowing that they have helped scientific advances, especially for families who are facing a similar medical issue.
What are the side effects of Pediatric Clinical Trials & Experimental Medication?
Each clinical trial or experimental medication has different side effects. Before agreeing to participate, you’ll talk to the study coordinator and investigator (the researcher overseeing the study) who run the clinical trial. They will answer all your questions about possible side effects.
At Children’s Health, we work with you to carefully weigh side effects and risks against potential benefits. After learning about the clinical trial opportunity for your child, you can decide whether you wish for your child to enroll in the trials.
What are the risks of Pediatric Clinical Trials & Experimental Medication?
The main risks of clinical trials are that your child will experience side effects. All the potential risks related to the clinical trial or experimental medication that we know of are outlined on a consent form. The study coordinator and investigator will go over this form with you. Then you and your child can decide if you want to join in the trial.
What to expect with Pediatric Clinical Trials & Experimental Medication?
Every clinical trial is different and follows different rules. Children’s Health is home to medical staff who specialize in guiding families through these trials and supporting them along the way.
Some clinical trials are relatively short. Others can last years. You can decide to stop participating in the trial at any time. Your decision, whether to participate, continue in the trial or discontinue at any point does not affect your child’s medical care.
Your involvement in a clinical trial is protected. Your child’s name and family information will never be made public.
What to expect before Pediatric Clinical Trials & Experimental Medication?
Before the clinical trial starts, you’ll be assigned a trial coordinator. Along with the study physician investigator, the trial coordinator will guide you through detailed explanations of the rules and procedures that your clinical trial will follow. Your trial coordinator will be available to answer any questions you have throughout the trial. Your doctor, who may also be the study’s physician investigator, will be by your side every step of the way, to answer questions and monitor how your child responds to the new treatment.
We'll collect what’s called baseline information for a month or two before we start your child on the medicine or in the control group. That way, we can compare what things looked like before and after the trial started.
You’ll also be asked to keep detailed records of how your child is doing. For example, if your child is feeling sick one day, you’ll be asked to write it down and describe what happened.
What to expect during Pediatric Clinical Trials & Experimental Medication?
The experience of being in a clinical trial can vary significantly, depending on your child’s health issue and what type of trial they’re in.
Overall, most families can expect to be in close contact with their study coordinators and care providers. You will probably need to come to the clinic more often, because we’ll be monitoring your child closely and collecting data that will help you and other families.
You can expect us to keep a close eye on your child, to see if the therapy is working and watch out for side effects.
What to expect after Pediatric Clinical Trials & Experimental Medication?
You’ll be asked to do a comprehensive exit visit at the end of your clinical trial. During this visit, we’ll do the same monitoring as we’ve been doing throughout the trial. We’ll compare the baseline information from when the trial started to where we are now. You’ll have a chance to ask any last questions.
Sometimes, your child may be able to continue using the medicine they were using during the study.
At some point after the trial ends (usually months or years), you may hear that the medicine is coming to market or that the FDA approved it for children.
How do I prepare my child for Pediatric Clinical Trials & Experimental Medication?
Let your child know that they will need to come to the doctor more often. We know this is inconvenient. But if your child realizes that participating in the trial could help other children like them, it can make it seem like less of a burden.
Also, if they are old enough, you can tell them they’ll be asked to sign an assent form to participate. This form says they understand that they are part of a trial and agree to participate.
What questions should I ask my provider about Pediatric Clinical Trials & Experimental Medication?
- What are the potential benefits?
- What are the risks?
- What are the side effects of this medicine?
- When will I know when my child's getting the medicine and not the control group?
Frequently Asked Questions
Does insurance cover experimental drugs and clinical trials?
Everyone who participates in a clinical trial is a volunteer. Some clinical trials are sponsored by organizations or companies. If your insurance doesn’t typically cover the cost of the medicine or other aspects of the trial, our team will work hard to find a way to make the trial free or affordable for you.
Are experimental drugs FDA approved?
Experimental drugs are often offered through clinical trials to explore whether the FDA should approve the drug.
In some cases, experimental drugs have already been FDA approved for older children or adults, or for children with other health conditions. Clinical trials can help determine if those drugs are safe and effective in younger children, or in children with other conditions.
Other times, the drug might be FDA approved, but the clinical trial is being done to learn more about the possible side effects or best dosage of that drug in young children.
