Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric Diffuse Intrinsic Pontine Gliomas (DIPG)
Running, not enrolling · Phase 1/Phase 2
Conditions studied: Brain Tumor
In brief
The purpose of this study is to evaluate the safety and efficacy of targeted blood brain barrier disruption with Exablate Model 4000 Type2.0/2.1 in combination with Doxorubicin therapy for the treatment of DIPG in pediatric patients
Key facts
- Study ID
- NCT05630209
- Run by
- InSightec
- People needed
- 10
- Starts
- 2023-01-04
- Expected to finish
- 2026-10-01
- Last updated by the study team
- 2026-04-17
Who can join
Age: 5 and older, up to 21. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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 it is running
- Children's National Medical Center — Washington D.C., District of Columbia, United States
- Nicklaus Children's Hospital — Miami, Florida, United States
- Cook Children's Health Care System — Fort Worth, Texas, United States
Full record on ClinicalTrials.gov
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