Using focused ultrasound and chemotherapy to treat pediatric brain tumors
A Safety and Feasibility Study to Evaluate Blood Brain Barrier Disruption Using Exablate MR Guided Focused Ultrasound in Combination With Doxorubicin in Treating Pediatric Patients With Diffuse Intrinsic Pontine Gliomas (DIPG)
This study is testing if using focused ultrasound along with chemotherapy can safely help children with a tough type of brain tumor called DIPG.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 5 Years to 21 Years |
| Sex | All |
| Sponsor | InSightec Industry-sponsored |
| Drugs / interventions | bevacizumab, trastuzumab, chemotherapy, immunotherapy, radiation, Doxorubicin |
| Locations | 2 sites (Washington, District of Columbia and 1 other locations) |
| Trial ID | NCT05630209 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and efficacy of using Exablate focused ultrasound to disrupt the blood-brain barrier in combination with Doxorubicin chemotherapy for treating pediatric patients with Diffuse Intrinsic Pontine Gliomas (DIPG). It is a prospective, single-arm, non-randomized feasibility study conducted at up to three sites in the United States. Participants will undergo three treatment cycles spaced approximately 4-6 weeks apart, with the aim of establishing the feasibility and safety of this combined approach and assessing its preliminary efficacy in this challenging patient population.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients aged 5 to 21 years diagnosed with DIPG who have completed radiation therapy and meet specific health criteria.
Not a fit: Patients with tumors not visible on imaging or those who have had recent brain surgery may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could improve treatment outcomes for children with DIPG by enhancing the delivery of chemotherapy directly to the tumor.
How similar studies have performed: While focused ultrasound is a novel approach in this context, similar studies have shown promise in other applications, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Washington, District of Columbia and 1 other locations
- Children's National Medical Center — Washington, District of Columbia, United States (Recruiting)
- Nicklaus Children's Hospital — Miami, Florida, United States (Recruiting)
Study contacts
- Study coordinator: Nadir Alikacem
- Email: nadira@insightec.com
- Phone: +12146302000
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.