What is a Clinical Trial?

Children with life-threatening conditions are often candidates for clinical trials. If your child has a serious illness, your doctor might talk to you about enrolling your child in a clinical trial.

Carefully conducted clinical trials are performed in human participants to provide answers to questions such as:

  • Does a treatment work?
  • Does it work better than the standard existing therapy?
  • Does it have side effects?

“Through clinical trials, doctors find new ways to improve treatments and the quality of life for people with disease. They also give children with rare or difficult-to-treat conditions, such as certain forms of cancer, access to innovative treatments,” says Dr. Van Huynh, a pediatric oncologist at the Hyundai Cancer Institute at CHOC Children’s, and the CHOC principal investigator for the Therapeutic Advances in Childhood Leukemia/Lymphoma Consortium, a group of children’s hospitals and universities that work to quickly develop and carry out clinical trials. The consortium develops phase I and phase II clinical trials of new therapies and novel therapy combinations in recurrent childhood leukemia and lymphoma.

dr-van-huynh-choc-childrens-pediatric-oncologist
Dr. Van Huynh, a pediatric oncologist at CHOC Children’s.

Clinical trials are standard practice in cancer treatment for children, adolescents and young adults. In fact, a higher percentage of newly diagnosed children with cancer and their families participate in clinical trials, perhaps more than in any other field of medicine, according to Dr. Huyhn. All clinical trials are voluntary and information collected is confidential. Participants can withdraw from a trial at any time, for any reason.

“Some clinical trials evaluate how well new medications work and whether they are safe to use. Other clinical trials explore whether we can use medications in new combinations or at different doses to improve survival or reduce short-term side effects,” says Winnie Stockton, investigational pharmacist at CHOC Children’s. “There are trials that focus on finding the best ways to minimize long-term side effects of certain treatments, and other trials that aim to determine the best dose to give children for medications that have successfully treated cancer in adults.”

winnie-stockton-choc-childrens-investigational-pharmacist
Winnie Stockton, an investigational pharmacist at CHOC Children’s

Types of Clinical Trials

Clinical trials are categorized as Phase I to IV trials.

Phase I

Studies of a new drug are the first that involve people. Participants in these trials are usually the first to receive the new therapy. The drug is tested to evaluate the dosages of the treatment and how often the treatment can be administered. Drugs are given at gradually increasing dosages, and participants are monitored closely for side effects. The goal of a Phase I clinical trial is to find the dose that is best tolerated without causing severe side effects.

Phase II

Once a treatment is found to be safe in a Phase I clinical trial, it is then tested in a Phase II trial to determine if it works. The treatment targets the patients who responded most favorably in Phase I trials. In these trials, doctors may evaluate if the treatment works for a specific type of cancer or illness.

Phase III

Once a new therapy has been determined to be safe and effective, it is then moved to a Phase III clinical trial. These are the studies that most children with newly diagnosed illnesses or cancers will receive. These trials test whether the new therapy is better than the standard treatment.

Phase IV (also known as Post-Marketing Surveillance Trials)

Phase IV studies take place after the drug has received regulatory approval (market authorization) and are designed to provide broader effectiveness and safety information about the new medicine in large numbers of patients, and to compare or combine it with other available treatments. These studies are designed to evaluate the long-term effects of the drug.

When you take part in a clinical trial, you will only be in that one phase of the study. Treatments move through the phases, but patients do not.

“At CHOC, we believe it’s important to conduct research directly with children and adolescents in a facility that is devoted exclusively to the care, quality of life, rights and safety of children of all ages. If a parent is interested in having their child participate in a clinical trial, we currently have over 350 research studies in more than 30 specialties to provide children with the latest treatments,” says Dr. Huynh.

Learn more about research at CHOC

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Research Leads to More Gratitude

As my 50-week gratitude tour for all things CHOC Children’s nears its end, I’d be remiss if I didn’t point out how grateful I am for the research that happens at CHOC.Research

CHOC is committed to clinical research and understanding the mysteries behind childhood diseases. Because of this, CHOC patients have access to current treatment options but also benefit from the latest in research.

Research is occurring every day at CHOC, and new endeavors begin regularly. Just recently, CHOC opened a stem cell production facility that could one day lead to therapies for neurological diseases in children.

The Research Institute at CHOC Children’s operates two Institutional Review Boards (IRBs), which decide whether proposed research is ethical, that participant consent is sufficient, and that safeguards have been established for participants.

It’s so exciting that CHOC is a research hospital. Scientists’ work today will affect patients for years to come. I am grateful that CHOC has an opportunity to help children today and tomorrow.

Childhood Vaccinations

To ensure maximum protection against diseases, children should receive vaccinations at the correct, recommended age, a CHOC Children’s infectious disease specialist tells Research 360,° a podcast highlighting research at the hospital.

In this segment, Dr. Antonio Arrieta discusses proper timing of vaccinations, dispels myths surrounding vaccinations, and details his current research at CHOC. Listen in to hear more about childhood vaccinations.

Hosted by Executive Director of Research Brent Dethlefs, Research 360° features interviews with scientists, physicians, educators, scientific news-and policy-makers to provide the listening audience with context, and scientific and social commentary intended to engage and inform.

Learn more about CHOC’s Research Institute.

CHOC Psychologist Discusses Pain in Children

It’s a misconception that children always outwardly express pain, and when they do, it’s often accomplished verbally and behaviorally, a CHOC Children’s psychologist tells Research 360,° a podcast highlighting research at the hospital.

In this segment, Michelle Fortier, Ph.D. says children experiencing pain may also exhibit changes in sleep patterns and eating habits, and may appear withdrawn. Listen below to learn more about pain in children, including how parents respond to pain, how different cultures view pain, and how to manage pain in children.

Hosted by Executive Director of Research Brent Dethlefs, Research 360° features interviews with scientists, physicians, educators, scientific news-and policy-makers to provide the listening audience with context, and scientific and social commentary intended to engage and inform.

Learn more about CHOC’s Research Institute.

The Institutional Review Board’s Role in Research

CHOC_IRB_ResearchBy Mellisa Henry, director of marketing, CHOC Children’s

As the marketing director at CHOC Children’s, I have a lot of different opportunities to participate in committees that may seem out of the normal marketing realm. One of the most interesting is having a seat on our Institutional Review Board (IRB) for clinical research.

Clinical research is used to determine the safety, dosing and effectiveness of medications, products and treatments used in prevention, diagnosis or treatment of various illnesses or medical conditions. It can range from immunizations to cancer to allergies and anywhere in between.

Any organization that is conducting clinical research is required by law to have its trial approved by an IRB. Organizations have the choice to hire an external IRB, or as CHOC chooses to do, structure an internal IRB. Committees must consist of members with both scientific and non-scientific expertise, and they have the duty to decide whether the proposed research is ethical, informed consent is sufficient, and that appropriate safeguards have been put in place for anyone participating in these trials.

And our responsibility weighs even heavier on my mind because we are acting on behalf of children.

As a non-clinician, I feel my responsibility is to look at the information with the eye of a mom. Do I understand what the research is trying to accomplish? Is the consent in language that allows me to understand? Do I understand what it means to the care of my child? What would my responsibility be in the process? And can I fulfill the responsibilities?

These may all seem like pretty basic questions, but the amount of information presented to a parent when their child is receiving care can be daunting. I want to ensure they can understand the information they receive without too much difficulty and make the choice that is right for their child and family.

Information gathered through clinical trials, and the advances in care that result, are incredible. I am forever in awe of the scientific minds that make these advances possible. But as a parent, it assures me to know that checks and balances are established through an IRB.

Related articles:

  • What is a Clinical Trial?
    Children with life-threatening conditions are often candidates for clinical trials. If your child has a serious illness, your doctor might talk to you about enrolling your child in a clinical ...
  • Research Leads to More Gratitude
    As my 50-week gratitude tour for all things CHOC Children’s nears its end, I’d be remiss if I didn’t point out how grateful I am for the research that happens ...
  • Childhood Vaccinations
    In this CHOC Radio segment, Dr. Antonio Arrieta discusses proper timing of vaccinations, dispels myths surrounding vaccinations, and details his current research at CHOC. Listen in to hear more about ...