Sound waves may help chemo reach deadly childhood brain tumors
NCT ID NCT05630209
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether a special ultrasound device can safely open the blood-brain barrier in children with a rare, aggressive brain tumor called DIPG. By temporarily allowing the chemotherapy drug doxorubicin to reach the tumor, researchers hope to improve treatment. The study involves 10 children aged 5 to 21 and focuses on safety and how well the barrier opens.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 10 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2023
- Expected to finish
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Oct 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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5 to 21 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age between 5 and 21 years, inclusive. Subjects younger than 5 years old may be eligible after discussion with the Sponsor Medical Monitor/designee (i.e. DSMB). * Patient diagnosed with DIPG * At least 4-week and not greater than 12 weeks from completion of radiation therapy * Post-radiation imaging does not show evidence of necrosis/ hemorrhage or other features that contraindicate MRgFUS * If brain surgery occurred, at least 14 days passed since last brain surgery and the patient is fully recovered and neurologically stable * If on steroids, stable or decreasing dose for at least 7 days prior to study entry * Stable or improving neurologic status for 7 days prior to study entry * Able and willing to give consent and/or assent or have a legal guardian who is able and willing to do so * Able to attend all study visits and with life expectancy of at least 6 months Exclusion Criteria: * Tumor not visible on any pre-therapy or post-radiation imaging * Previous participation in other chemotherapy, molecularly targeted therapy, or immunotherapy treatment-related phase 1 or 2 trials * Symptoms and signs of increased intracranial pressure * Subject with metastatic disease * Subject with ventricular peritoneal shunt * Subject receiving bevacizumab (Avastin) therapy or increasing doses of steroids * Anti-coagulant therapy, or medications known to increase risk of hemorrhage, (e.g., ASA, non-steroidal anti-inflammatory drugs \[NSAIDs\], statins) within washout period prior to treatment * History of a bleeding disorder, coagulopathy or with a history of spontaneous tumor hemorrhage. * Hypertension per age * Cerebral or systemic vasculopathy, including intracranial thrombosis, vascular malformation, cerebral aneurysm, or vasculitis * Immunosuppression (corticosteroids to prevent/treat brain edema are permitted). * Patients with positive HIV status * Active seizure disorder or epilepsy (seizures despite medical treatment) for a minimum of 4 weeks prior to first cycle/Exablate BBBD procedure captured by history * Known sensitivity to gadolinium-based contrast agents * Known sensitivity to DEFINITY® ultrasound contrast agent or known hypersensitivity to perflutren microsphere or its components, e.g., polyethylene glycol * Contraindication to Doxorubicin * Previous treatment with complete cumulative doses of Doxorubicin, daunorubicin, idarubicin, and/or other anthracyclines and anthracenediones * Severely impaired renal function with estimated glomerular filtration rate \<2 standard deviations for age * Patients that may require trastuzumab during the study * Patients that may require inhibitors and inducers of CYP3A4, CYP2D6, and/or P-gp during the study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Cook Children's Health Care System
Fort Worth, Texas, 76104, United States
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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Other studies related to the condition(s) this trial covers.
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