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 specific type of brain tumor called DIPG.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 5 Years to 18 Years |
| Sex | All |
| Sponsor | InSightec Industry-sponsored |
| Drugs / interventions | bevacizumab, chemotherapy, immunotherapy, radiation, Doxorubicin |
| Locations | 1 site (Toronto, Ontario) |
| Trial ID | NCT05615623 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and efficacy of using Exablate focused ultrasound technology 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 a single center in Canada. Participants will undergo three treatment cycles spaced approximately 4 to 6 weeks apart, aiming to establish the feasibility and safety of this combined treatment approach while assessing preliminary efficacy.
Who should consider this trial
Good fit: Ideal candidates are children aged 5 to 18 years diagnosed with DIPG who have recently completed radiation therapy.
Not a fit: Patients with evidence of cranial or systemic infection or those with contraindications to Doxorubicin may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could improve treatment outcomes for pediatric patients with DIPG by enhancing drug delivery 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 18 years, inclusive * 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 * Able to attend all study visits and with life expectancy of at least 6 months * Able and willing to give consent and/or assent or have a legal guardian who is able and willing to do so * If on steroids, stable or decreasing dose for at least 7 days prior to study entry * If brain surgery occurred, at least 14 days passed since last brain surgery and the patient is fully recovered and neurologically stable Exclusion Criteria: * Evidence of cranial or systemic infection * Known life-threatening systemic disease * Previous treatment with complete cumulative doses of Doxorubicin, daunorubicin, idarubicin, and/or other anthracyclines and anthracenediones * Contraindication to Doxorubicin. - Known sensitivity to DEFINITY® ultrasound contrast agent or known hypersensitivity to perflutren microsphere or its components, e.g., polyethylene glycol. - Known sensitivity to gadolinium-based contrast agents * 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 * Patients with positive HIV status. - Immunosuppression (corticosteroids to prevent/treat brain edema are permitted) * Cerebral or systemic vasculopathy, including intracranial thrombosis, vascular malformation, cerebral aneurysm, or vasculitis * Hypertension per age * History of a bleeding disorder, coagulopathy or with a history of spontaneous tumour hemorrhage * 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 * Patient receiving bevacizumab (Avastin) therapy or increasing doses of steroids * Symptoms and signs of increased intracranial pressure * Previous participation in other chemotherapy, molecularly targeted therapy or immunotherapy treatment-related phase 1 or 2 trials * Tumor not visible on any pre-therapy or post-radiation imaging
Where this trial is running
Toronto, Ontario
- Sunnybrook Research Institute — Toronto, Ontario, Canada (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.