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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Looking beyond traditional medicine

Guest blogger, Audra Wilford, proud mom of CHOC Children’s patient, “SuperMax,” and founder of MaxLove Project – a community organization dedicated to helping childhood cancer families beat the odds – shares how her son has benefitted from CHOC’s integrative health services.

Max with Ruth McCarty, director of CHOC's Integrative Medicine Program
Max Wilford with Ruth McCarty, L.Ac.

A few years ago, our son Max was diagnosed with brain cancer. After a difficult brain surgery, where only a portion of the tumor could be removed, and almost a month in the hospital, we faced the biggest challenges of our lives. After the surgery, Max lost his ability to walk, talk and use the left side of his body. He was going to need an unknown amount of chemotherapy and radiation to try to shrink the tumor left in his brain. We knew we were going to need an all-hands-on-deck approach for fighting the cancer and for restoring Max to health.

What we didn’t realize at the time was how many hands we’d need – it takes a whole multi-disciplinary team. But what happens when we leave the hospital? Who helps with nagging side effects, lingering stress, optimal nutrition, strengthening exercises and healthy sleep?

For us, it started with one amazing CHOC practitioner: Ruth McCarty. Ruth is the country’s only traditional Chinese medicine specialist at a children’s hospital who is fully integrated into every medical team in the hospital. When we first met with her, she put us at ease immediately. As we told her about Max’s neuropathy, sleep problems, headaches, gut challenges, anxiety, balance problems, muscle pain, immune suppression, and so on, she smiled and told us not only what she could do with acupuncture and moxibustion – a Chinese medicine technique that involves the burning of mugwort, a small spongy herb, to facilitate healing – but also what we could do at home through massage and select supplementation.

Over the next few months we saw Ruth every week, twice per week during chemotherapy. Max’s strength, range of movement, mood, pain, gut challenges, headaches, and sleep improved every week. And on the rare week when we had to miss appointments with Ruth, we would notice symptoms returning.  During this time we also got to know Dr. Agnes Horvath, a CHOC oncologist who made the rounds in the outpatient infusion center. Dr. Horvath introduced us to bone broth and other healing foods that helped Max thrive through treatment. She encouraged us to think about nutrition as a key component of Max’s treatment. With her help, we continued to transform Max’s diet into what we came to call “fierce foods”: lots of whole foods, limited sugar and processed foods, and a ton of flavor. Max finished treatment in January 2013 and finished up kindergarten just a few months later.

By June 2013, Max’s tumor began growing again. By then, we had become immersed in the latest research on nutrition and cancer, and we wanted to try an intensive therapeutic diet for Max alongside whatever conventional therapies that our oncologist, Dr. Violet Shen, recommended. Dr. Shen took the time to research the diet and gave us her full support. She carefully guided us as we incorporated a therapeutic diet into Max’s treatment protocol.

Today, Max’s tumor continues to shrink and he’s a healthy, happy third grader. Each step of the way, CHOC has supported our family in incorporating the best and latest evidence-based medicine, whether it’s in acupuncture, massage therapy, stress control or diet. The doctors, nurses and therapists at CHOC understand that complementary and integrative medicine is about optimum quality of life, using the best of all therapeutic approaches to support each child and family to thrive in the face of tough odds.

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Innovative Cancer Treatment: Abbey’s Story

Abbey and Jaime Serna, phlebotomist at CHOC Children's hospital.
Abbey Schemmer and Jaime Serna, phlebotomist at CHOC Children’s Hospital.

While many teenaged girls might balk at wearing a medical device on their shaved heads 18 hours a day, Abbey Schemmer didn’t blink an eye.

After all, the 16-year-old CHOC Children’s patient was fighting for her life.

Following the diagnosis of two brain tumors – including a rare, extremely aggressive type called glioblastoma multiforme – and several surgeries, Abbey and her family turned to a new technique to halt the tumors’ growth: tumor treating fields, or TTF.

TTF uses electromagnetic forces to prevent this type of tumor from dividing and growing. TTF was only recently approved for adult use, and as a pediatric patient, Abbey received special clearance to use ahelmet-like device that administers the treatment.

Worn continuously for 18 hours a day, the helmet places electrodes directly on Abbey’s scalp, which requires her to shave her head every few days so that the helmet’s electrodes directly touch her skin. Abbey wears the device’s power source in a backpack.

“This is not an easy treatment,” said Dr. Violet Shen, Abbey’s oncologist at CHOC. “But she was willing to do that and is wearing her helmet and head dressings and still going on with her life.”

Following about nine months of TTF treatment and chemotherapy, Abbey’s tumor shows no signs of major progression, Dr. Shen said.

The progress is a marked departure from expectations when Abbey was diagnosed in early 2013 following complaints of nausea, headaches and lethargy.

“We thought we had a sick little girl,” said Ken, Abbey’s father. “We were thinking she wouldn’t live for very long.”

Abbey immediately underwent surgery to remove the tumor, followed by chemotherapy and radiation. She stayed in good spirits and continued with school, but after she experienced seizures in summer 2014, it was clear the glioblastoma had grown back.

After another surgery to remove Abbey’s right frontal lobe, the family explored other methods to curb the tumor’s growth, and ultimately turned to TTF.

“It was cool that it could help my cancer,” Abbey said.

In the months following, Abbey has continued in school and she’s looking forward to beginning driver’s education. Her schoolmates have rallied around her, and taken an interest in her unique treatment, Abbey said. “People ask me questions,” she said. “They think it’s fun because my backpack generates heat. They ask, ‘Can I see your heater?’”

If this treatment continues to work, Abbey could feasibly continue to wear the helmet for the rest of her life, Ken says. For now, her family is taking the treatment one day at a time.

“It’s been working,” Dr. Shen said. “Very few people survive with this tumor for six months, let alone a year. Abby’s done well. She’s very vigorous and still attending school. Abbey’s definitely a fighter.”

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Celebrate National Cancer Survivors Day

In honor of National Cancer Survivors Day on June 7, check out this video where patients and staff at the Hyundai Cancer Institute at CHOC Children’s groove to Lady Antebellum’s “Compass” and show how they let their hearts be their compasses.

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  • Looking beyond traditional medicine
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  • Innovative Cancer Treatment: Abbey’s Story
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Bringing Together the Best Minds in Pediatric Cancer Care

CHOC Children’s is taking the concepts of second-opinion consultation and tumor board planning at least six steps further. The Virtual Pediatric Network (VPN) uses state-of-the-art video conferencing to allow pediatric cancer experts from six leading institutions to share best practices, research and expertise as if they were all in the same room.

“No one institution can be an expert in every cancer, but with the VPN, these hospitals can work together to bring the best care to patients wherever they may live,” said Dr. Leonard Sender, medical director, Hyundai Cancer Institute at CHOC.

Described as a “multidisciplinary second opinion,” the tumor board concept has become an important cancer-fighting resource at hospitals in recent years. Tumor board meetings typically involve staff from across medical, nursing, psychological and rehabilitative disciplines who evaluate, diagnose and plan treatment for individual patients.

In a similar fashion, the VPN is comprised of five medical centers and one research institute for genomic medicine: CHOC, Lucile Packard Children’s Hospital at Stanford, Kapi’olani Medical Center for Women & Children, Duke Children’s Hospital & Health Center, Rady Children’s Hospital-San Diego and the Translational Genomics Research Institute. With CHOC serving as the hub, all are connected through the CISCO TelePresence video conferencing system, a secure network made available by a grant from CISCO to the CHOC Children’s Foundation.

According to Dr. Sender, the TelePresence system offers the highest level of video conferencing technology available. Optimized for exceptional sound and picture quality, the system captures every expression, gesture and voice inflection, creating a collaborative environment that leads to deeper discussion, enhanced information sharing and improved decision making.

“The VPN is an opportunity to leverage technology to allow for greater collaboration and, hopefully, innovation between children’s hospitals and cancer programs,” Dr. Sender said. “This will lead to potentially better science and provide pediatric patients the greatest opportunities for survival and quality of life.”

The VPN has the potential to standardize and improve care for pediatric patients throughout the world, Dr. Sender added. CHOC and CISCO are already planning to expand the network to additional locations and specialties, including pediatric institutions in Australia and the United Kingdom.

Learn more about the Hyundai Cancer Institute at CHOC Children’s.

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