Research conducted with children has unique challenges. Seemingly simple steps like obtaining consent, taking vital signs, placing IVs and drawing blood can become very difficult.
And it only takes one bad research experience to sour a child’s outlook or a family’s enthusiasm to participate.
|Burt Nabors, left, director of PCIR, and J.P. Clancy, director of CHRU, are charged with restructuring UAB’s research enterprise to facilitate translational science in ways that improve the overall health of the population and reduce health disparities.
The UAB Center for Clinical and Trans-lational Science (CCTS) recently partnered with the UAB Department of Pediatrics and Children’s Hospital of Alabama to develop the Child Health Research Unit (CHRU) to better engage and care for children participating in research. The partnership with Children’s Hospital gives UAB physicians and researchers a child-friendly environment and access to staff with the expertise to better address the physical and emotional needs of children.
The CHRU will enable researchers to better conduct clinical trials, simplify participation for the study subjects, consolidate pediatric research in one site and provide access to pediatric services not generally available in an adult facility.
“This is advantageous for our patients, physicians and researchers because the services provided by Children’s Hospital can really help to make the research process go smoothly,” says J.P. Clancy, M.D., director of the CHRU. “You’re able to see study subjects in a research-dedicated space and access services like IV placement, radiology and lab services by experts in the care of children.”
The CHRU, located on the seventh floor of Dearth Tower in Children’s Hospital, is structured much like the adult Participant and Clinical Interactions Resources (PCIR), another component of the CCTS. Both provide space, access to nursing and lab processing for investigators.
Clancy says the goal of the CHRU is to create a platform for investigators to conduct research with pediatric subjects that is safe and flexible to accommodate the needs of the investigator and the study participants.
“I believe that we have a great opportunity to create research relationships across departments and schools at UAB, especially when you can partner that with the great facilities and expertise available within Children’s Hospital,” Clancy says.
Pivotal trials under way
Phase II and III trials of new drugs to treat the underlying cause of cystic fibrosis (CF) currently are under way in the CHRU.
The drugs, known as CFTR modulators, target the defective protein that causes CF. The approach is considered unique because all other therapies used for CF target downstream symptoms and not the root cause of the disease.
“This is not gene therapy in which you replace a gene,” says Clancy, the principal investigator of the international Phase II study of VX-809, a small molecule designed to help correct the most common cause of CF. “This uses a drug in pill form to help your own gene or gene product work better.”
UAB is playing a key leadership role in the VX-809 trial and the Phase III international trial of PTC124, a study drug intended to restore function to CF-causing mutations. Steven Rowe, M.D., assistant professor of medicine, is the co-principal investigator of the drug PTC124, which is a drug that grew out of research performed by David Bedwell, Ph.D., in microbiology, and the UAB Gregory Fleming James Cystic Fibrosis Research Center more than 10 years ago.
“These are first-in-class drugs,” Clancy says. “No one has ever tried them before.”
Researchers (including UAB) recently completed a trial of a related drug known as VX-770, which targets a different error in the gene that causes CF. The results of that study were very promising and have been reported at international meetings during the past six months.
“There were large improvements in lung function, and we were able to detect restored activity to the CF gene product in VX-770 treated patients,” Clancy says. “We’re still blinded on VX-809 and PTC124, so we don’t know the results yet, but we are optimistic.”
Other areas of research already are taking place, and Clancy says it is likely that nutrition-based studies will be implemented rapidly. “This has been an established area of expertise in the UAB Department of Nutrition Sciences for a number of years,” Clancy says.
PCIR available to UAB researchers
The CHRU was created to mirror and complement the PCIR, which was known formerly as the General Clinical Research Center.
The adult-service PCIR is involved primarily in outpatient research, but does have inpatient research capabilities.
The PCIR is open to investigators throughout campus Monday through Friday from 6:30 a.m. to 6 p.m. and Saturday as needed.
“If a study has a need for us to assist with the administration of a drug, infusion of a drug or collection of pharmacokinetic samples, we can do it,” says Burt Nabors, M.D., director of the PCIR. “We provide nursing, bio-nutrition care and space and can provide care for patients that need to be hospitalized for the type of research study they are involved in. We do many nutrition studies and metabolic work with researchers and nutrition. We have a pretty broad capability.”
The PCIR is located on the 15th floor of Jefferson Tower. It is undergoing renovations that will enhance and centralize the metabolic kitchen and expand and update the patient-care areas.
The PCIR and the CHRU are both components of the CCTS, a research enterprise created from the five-year, $26.9 million Clinical and Translational Science Award from the National Institutes of Health. Its charge is to restructure UAB’s research enterprise to facilitate translational science in ways that improve the overall health of the population and reduce health disparities.
“The CCTS is strongly committed to developing the highest quality platform for the effective and efficient performance of clinical and translational research that impacts the lives of children and adults,” says Lisa Guay-Woodford, M.D., director of the CCTS. “We are extremely grateful to the Children’s Health System for their support in developing the CHRU. This resource is a critical component of our infrastructure and will be essential in helping us achieve the goals of our center.”
Learn more about the PCIR and download an application for PCIR services at www.ccts.uab.edu/pages/pcir.aspx. Visit www.ccts.uab.edu/pages/pcir_CHRU.aspx to learn more about the CHRU.