Key Points
- A clinical trial investigating focused ultrasound to treat benign pediatric brain tumors has been extended for children with hypothalamic hamartomas.
- This extension is being funded by the Foundation, Hope for Hypothalamic Hamartomas, and Insightec.
The first 5 of 10 additional patients have been enrolled in the extension of a pioneering clinical trial (NCT03028246) investigating focused ultrasound as a treatment for benign pediatric brain tumors. The extension is being funded by the Foundation in partnership with Hope for Hypothalamic Hamartomas and Insightec.
The original clinical trial, launched at Nicklaus Children’s Hospital in Miami in 2017, evaluated the use of Insightec’s Exablate Neuro focused ultrasound device to ablate centrally located benign brain tumors in children and young adults ages 8 to 22. Ten participants were treated in the initial phase of the study at two sites – Nicklaus Children’s Hospital and Children’s National Hospital in Washington, DC.
The trial enrolled patients with a range of benign tumors, including hypothalamic hamartomas, pilocytic astrocytoma, and subependymal giant cell astrocytoma (SEGAs). Researchers sought to demonstrate that the incisionless therapy could safely target lesions that can cause seizures, cognitive impairment, and other neurological complications while avoiding many of the risks associated with conventional brain surgery.
In 2022, early results from the first five patients were published in the Journal of Neurosurgery. The procedure was safe, well tolerated, and free from complications. Ablation was successful in four participants with hypothalamic hamartomas and led to improved seizure control and decreased hyperphagia.
Extending the trial to enroll 10 additional patients will generate a larger body of evidence on the role of focused ultrasound in pediatrics. Researchers expect the results from the first cohort of patients to be published by the fall of 2026. The extended trial is taking place at Nicklaus Children’s Hospital in Miami, Children’s National Hospital in Washington, DC, Texas Children’s Hospital in Houston, and the Children’s Hospital of Philadelphia. The principal investigator is John Ragheb, MD, chief of the Department of Surgery at Nicklaus Children’s Hospital.
“I am very optimistic that this current trial in children with hypothalamic hamartoma will confirm the promising seizure control and the quality of life improvement observed in the initial study,” said Dr. Ragheb.
“Individuals and families affected by hypothalamic hamartomas have long struggled with more invasive surgical options that have often resulted in endocrine and short term memory issues,” said Lisa Soeby, MBA, vice president and co-founder of Hope for Hypothalamic Hamartomas. “Having a noninvasive radiation free option that shortens recovery time and reduces potential complications is huge for our community!”
For Patients
For more information about this clinical trial, please contact the following sites:
Nicklaus Children’s Hospital
305-662-8386
mrguidedfus@Nicklaushealth.org
John Ragheb, MD
305-662-8386
john.ragheb2@nicklaushealth.org
Children’s National Hospital
Elizabeth Paronett
202-476-5551
eparonett@childrensnational.org
Texas Children’s Hospital
Sarah Martinez
832-822-4075
sxwisor@texaschildrens.org
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