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Clinical Trials in the USA / NCT05630209
Active, not recruiting Phase 1/2

Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric Diffuse Intrinsic Pontine Gliomas (DIPG)

NCT05630209 · tracked via the Priya Life Science USA tracker
Sponsor
InSightec
Phase
Phase 1/2
Started
2023-01-04
Last updated
2026-04-17

Condition(s) studied

Brain Tumor

Investigational drug(s) / intervention(s)

ExablateDoxorubicin

Exablate: Blood Brain Barrier Disruption (BBBD) via Exablate Type 2 system with microbubble resonators on the day of Doxorubicin infusion to treat DIPG brain tumors

Doxorubicin: Doxorubicin infusion

Study summary

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

Eligibility

Sex
ALL
Min age
5 Years
Max age
21 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (3)

FacilityCityRegionStatus
Children's National Medical Center Washington D.C. District of Columbia
Nicklaus Children's Hospital Miami Florida
Cook Children's Health Care System Fort Worth Texas

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05630209 on ClinicalTrials.gov ↗ ← All trials in the USA