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

Launched by INSIGHTEC · Nov 7, 2022

Keywords

ClinConnect Summary

This clinical trial is looking at a new way to treat a type of brain tumor called DIPG (Diffuse Intrinsic Pontine Glioma) in children and teenagers aged 5 to 18. The study aims to see if a special technique called focused ultrasound, combined with a chemotherapy drug called Doxorubicin, can safely help treat this challenging condition. The researchers want to find out if this method can disrupt the blood-brain barrier—a protective layer that makes it hard for treatments to reach the tumor—allowing the medication to work better.

To participate in the trial, patients must be diagnosed with DIPG and have completed radiation therapy within the last 4 to 12 weeks. They should be able to attend all study visits and have a life expectancy of at least six months. It's important that they are stable and healthy enough for the study, meaning they should not have any serious infections or other health issues that could complicate treatment. Participants can expect to undergo monitoring and assessments during the trial to ensure their safety and to evaluate how well the treatment is working. This study is currently recruiting participants, and families interested in this option should talk to their healthcare provider for more information.

Gender

ALL

Eligibility 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