Cook Children's is at the forefront of clinical research

We leave no stone unturned to help out-of-state and international families find the latest therapies for their child.

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Pediatric Research Opportunities for Out-of-State and International Patients

Cook Children's excels not only in first-class clinical care but also in leading-edge pediatric research. Our growing portfolio of studies provides children access to today's most innovative therapies.

90 investigators

427 studies

10550 participants

Knowing every child's life is sacred, it's our promise to improve the well-being of every child in our care and our communities. Research allows Cook Children's to fulfill this promise through large clinical trials and with studies consisting of just one child.

William Stigall, M.D. – Cook Children’s Chief Research Officer

Research specialties

Cook Children's researchers have a deep desire to understand childhood diseases and find better ways to diagnose, treat and prevent them. With more than 400 active research projects and new ones opening in nearly every program, our reputation is growing.

Some of the leading areas of research at Cook Children's are:

Pediatric cancer

Children with cancer often receive their treatment as part of a clinical trial. The main groups coordinating pediatric cancer clinical trials in the U.S. are Children's Oncology Group and St. Jude Children's Research Hospital.

Cook Children's is one of the few centers that participates with both groups. Because of this participation, we can offer nearly any experimental treatment available today, such as:

CAR T-cell therapy, a type of cellular immunotherapy for blood cancers
I-131 metaiodobenzylguanidine (MIBG) therapy, targeted therapy and small molecule immunotherapy for neuroblastoma
New therapies for brain tumors

With different treatment protocols to choose from, we can personalize care for each child's needs. Researchers are also looking beyond treatment to improve access to care, survivorship support and psychosocial services for adolescents and young adults. Learn more about hematology and oncology research.

Neurological diseases

The Jane and John Justin Neurosciences Center is at the forefront of pediatric neuroscience research with an emphasis on epilepsy and movement disorders. Highlights of our work, which merges the latest technological advances and evidence-based care, include:

Epilepsy

Our epilepsy research program is among the most advanced in the country. We are investigating advanced neuroimaging tools and techniques to identify where seizures start. As a member of the Pediatric Epilepsy Research Consortium, we are helping identify best practices for evaluating and treating epilepsy.

Scott Perry, MD, head of the Jane and John Justin Institute for Mind Health, is one of the world's top epileptologists. Dr. Perry seeks to improve surgical therapies to cure epilepsy. He also founded Cook Children's Genetic Epilepsy program, which is pioneering therapies for rare genetic epilepsies, such as Dravet syndrome and Lennox-Gastaut syndrome.

Movement disorders

Cook Children's researchers are helping improve the care for children with movement disorders, including:

Dystonia: We are investigating multi-modal brain imaging to assess functional changes induced by deep brain stimulation treatment.
Cerebral palsy: We are using advanced rehabilitation methods, such as pediatric robotic therapy, to treat functional deficits of the upper extremities.

Endocrinology

Cook Children's Hyperinsulinism Center is one of only two U.S. Centers of Excellence and one of six worldwide. Led by internationally recognized expert Dr. Paul Thornton, we are helping cure many patients with congenital hyperinsulinism.

Treating congenital hyperinsulinism used to require removing the entire pancreas, which resulted in diabetes. For patients with focal hyperinsulinism, we can identify and remove the area making too much insulin. This innovative treatment uses an investigational type of imaging — an 18F DOPA PET-CT scan. Successful care relies on extensive expertise to interpret the scans and develop a treatment plan.

Other endocrinology research initiatives include:

Screening and assessment for cardiovascular risk factors
Weight management in children and adolescents

Pulmonology

In 2009, Cook Children's became a Therapeutic Development Center (TDC) through the Cystic Fibrosis Foundation. As a TDC, we are part of a network of centers investigating aspects of cystic fibrosis treatment, including:

Long-term surveillance of patients with cystic fibrosis
Pulse oximetry overuse
Therapeutic interventions for cystic fibrosis

Bone marrow and stem cell transplantation

As one of the most experienced pediatric transplant programs in the Southwest, we actively research to:

Determine the best candidates for bone marrow and stem cell transplant
Benchmark outcomes with academic and international transplant centers
Understand the needs of the transplant patients

Genetics

Our emerging Pharmacogenomics Program is investigating how to use genetics to select the most effective medications. Areas of focus for this work include:

Hematology and Oncology
Genetics
Immunology
Psychiatry

Initiating single-person studies

Research studies are not always available when a patient needs an investigational drug. In some cases, a drug is available outside of a study but is prohibitively expensive. That's when Cook Children's promise of “everything for the child' makes a difference.

Our research specialists work with drug manufacturers and the FDA to open single-patient studies when possible. This process is complex and requires extraordinary effort from our experienced staff. Since 2011, we've helped more than 60 children get the drugs they need, saving their families millions of dollars in drug costs.

Cook Children's has also formed a new partnership with the n-Lorem Foundation to open single-patient studies for patients with nano-rare conditions. These conditions affect less than 30 patients worldwide. The n-Lorem Foundation develops therapies for these uncommon conditions, obtains FDA approval and helps oversee lifelong patient care.

Comprehensive support for referring physicians and families

Traveling for a child's medical care can be challenging for families who live in another state or country. Our research and Global Health Services teams help make the process easier and less stressful by streamlining:

Care coordination

We work closely with referring providers throughout the care journey:

Before treatment: We determine what tests and treatments your patient can complete locally. This helps reduce travel and costs.
During treatment: We provide updates on your patient's condition, their treatment and who to call with questions.
After treatment: We work with you to make the transition as smooth as possible when it's time to transfer care back to you.

Billing and insurance

Investigational drugs are usually free for patients enrolled in clinical trials, but other medical costs may not be. Our researchers, Global Health Team and utilization review specialists work together to estimate out-of-pocket costs and find ways to reduce those costs.

Travel

Our Global Health specialists help families navigate travel to Fort Worth, Texas, and find accommodations, food and recreation while here.

Translation

It's essential to make sure families understand the research protocol, consent process and every aspect of their child's care. We use certified translators and translation services to help us communicate with families in their language.

Why refer your patient to Cook Children's for pediatric research?

Cook Children's researchers have spent years, sometimes decades, investigating childhood diseases within their areas of expertise. Physicians and families across Texas and beyond turn to us for our experience and compassionate approach to care.

When you refer a child to us for a research study or clinical trial, you and your patient's family can expect:

Specialized expertise: Cook Children's is home to world-renowned researchers transforming their fields - and children's lives. They participate in investigator-initiated research and sponsored research through pharmaceutical companies and collaborative groups.
Dedicated team: Coordinating research activities across Cook Children's requires a large team of specialists. Our Research department includes nurse coordinators, clinical research coordinators, clinical data managers, PhD and master's level scientists, regulatory experts and pharmacists.
Personalized approach: Research studies may have specific criteria for enrolling participants. For children who are not eligible for a study, we work hard to find other options. We may be able to open a single-person study for an investigational drug if a clinical trial is not available. Investigational drugs are in development but have not received Food and Drug Administration (FDA) approval. They are only an option for serious or life-threatening illnesses when other effective treatments are not available.
Safety and quality assurance: We have many protections in place to ensure children's safety. All studies have a carefully designed protocol and receive annual approval from the Cook Children's Institutional Review Board. Our Regulatory Affairs division and Investigational Drug Services Pharmacy closely monitor research activities.
Full-service support: Our research and Global Health Services teams help physicians and families navigate a child's care. We provide support and guidance at every step, from referral to travel, billing and returning home.

Refer a patient

Referring physicians typically work directly with a researcher to enroll patients in a specific trial.

Find active research projects

For more information or assistance, please contact:

Clinical Research department at 01-682-885-2103 or CookChildrensResearch@cookchildrens.org.
Global Health Services at 01-682-303-1376or GlobalHealthServices@cookchildrens.org.

All you have to do is reach out. We'll handle the rest.