Duke & UNC Researchers to co-lead $12.9M Study to Improve Care for Autistic Children
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DURHAM, N.C. -- Duke University has been awarded $12.9 million from the Patient-Centered Outcomes Research Institute (PCORI) for a new clinical trial aimed at improving the standard of care for autistic children with chronic digestive discomfort.
The study will be co-led by researchers at UNC-Chapel Hill, underscoring the impact of the partnership between Duke Health and UNC Health to form North Carolina Children’s, the state’s first freestanding children’s hospital, which is planning to break ground on its Apex, N.C., campus in 2027.
Pediatric Gastroenterologists Bruno Chumpitazi, M.D., of Duke and Michael Kappelman, M.D., of UNC will serve as co-principal investigators for the study, with the Duke Clinical Research Institute serving as the site and data coordinating center, and the UNC Patient and Community Engagement in Research Program serving as the patient engagement hub.
Addressing a Gap in Care
Up to 65% of autistic children experience constipation, a rate that is three times higher than the general population. The condition is associated with irritability, social withdrawal, disruption of daily routines, and even self-injury.
“We see firsthand that standard treatments don’t always work well for autistic children, where sensory sensitivities, diet, and medication tolerance can complicate care,” said Chumpitazi, a professor in the Department of Pediatrics at the Duke University School of Medicine. “This study is designed to generate clear, evidence-based guidance that clinicians and families can use.”
Treatment recommendations for autistic children are the same as for the general population, even though the current standard of care is not always tolerated or effective in autistic youth.
“For autistic children, constipation is associated with a higher number of emergency department visits and hospitalizations. Determining better evidence-based treatments may make a major difference,” said Kappelman, professor in the Department of Pediatrics at the UNC School of Medicine.
Similarly, adherence and tolerance to medications commonly used to treat constipation can be challenging for autistic youth due to issues related to texture and volume.
Clinical Trial with Broad Implications
The multi-site, randomized, head-to-head clinical trial will compare the effectiveness of linaclotide to the current standard treatment, polyethylene glycol 3350 (PEG-3350, commonly known by the brand name MiraLAX®) for treating constipation in autistic children.
The study team aims to recruit 320 patients from 30 sites across the nation, drawing from general pediatrics, developmental pediatrics, and pediatric gastroenterology practices at large freestanding children’s hospitals and smaller hospitals and clinics. Patients and parents will be integral members of the study team across the project.
Because this will be the first clinical trial to directly compare these treatment options, researchers believe the results may also have implications for the broader population of children who suffer from constipation.
Collaboration Through NC Children’s
Beyond the clinical impact of this research, this study reflects the strength of the NC Children’s partnership.
“This is a wonderful collaboration between our two pediatric programs and a great example of what we can accomplish together through NC Children’s,” said Kappelman.
This study addresses a pressing need for clearer, safer treatment pathways, and its findings are expected to support parents, guide clinicians, and strengthen the collaborative care that defines NC Children’s mission and goals of creating a world-class children’s hospital.
“This study reflects the kind of collaboration we are building through NC Children’s—bringing together Duke’s research infrastructure and UNC’s patient engagement expertise to improve care for children across the country,” said Geeta Swamy, M.D., NC Children’s Chief Academic Officer, who also serves as Executive Vice Dean for Clinical Sciences & Research Administration for the Duke University School of Medicine and Associate Vice President for Research for Duke University.
The funding to Duke University has been approved pending completion of PCORI’s business and programmatic review and issuance of a formal award contract.