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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05615623
Recruitment Status : Recruiting
First Posted : November 14, 2022
Last Update Posted : January 6, 2023
Sponsor:
Information provided by (Responsible Party):
InSightec

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety and efficacy of targeted blood brain barrier disruption with Exablate Model 4000 Type 2.0/2.1 in combination with Doxorubicin therapy for the treatment of DIPG in pediatric patients

Condition or disease Intervention/treatment Phase
Brain Tumor Device: Exablate Drug: Doxorubicin Phase 1 Phase 2

Detailed Description:
This is a prospective, single arm, non-randomized feasibility study to evaluate the safety, feasibility and preliminary efficacy of Blood Brain Barrier Disruption (BBBD) using the Exablate Type 2 system in pediatric patients with Diffuse Intrinsic Pontine Gliomas (DIPG) undergoing Doxorubicin chemotherapy. The study will be conducted at a single center in Canada. Patients will undergo 3 treatment cycles, approximately 4 -6 weeks apart. The study aims to establish feasibility and safety of Exablate BBBD in conjunction with Doxorubicin in the treatment of pediatric DIPG and assess preliminary efficacy in this patient population.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 3 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Device Feasibility
Official Title: 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)
Actual Study Start Date : January 4, 2023
Estimated Primary Completion Date : January 4, 2025
Estimated Study Completion Date : July 4, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Blood Brain Barrier Disruption (BBBD)
Exablate MR Guided Focused Ultrasound for Blood Brain Barrier Disruption with Doxorubicin for treating pediatric patients with DIPG
 Device: 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
Other Name: Exablate BBBD

Drug: Doxorubicin
Doxorubicin infusion
Other Names:
  • Adriamycin
  • Rubex


Outcome Measures
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Primary Outcome Measures :
  1. Adverse Events [ Time Frame: 2 years ]
    All adverse events from first treatment to end of study will be documented and reported according to the CTCAE terminology and severity scale


Secondary Outcome Measures :
  1. Blood Brain Barrier Disruption (BBBD) [ Time Frame: Immediately post BBBD sonication ]
    BBBD will be assessed as a comparative ratio measured in pre and post-sonication contrast-enhanced MR images


Other Outcome Measures:
  1. Tumor Volume [ Time Frame: Immediately post BBBD sonication ]
    Average volume of contrast-enhancement on post sonicated MRI scans will be compared with pre-sonicated scans

  2. Progression Free Survival (PFS) and Overall Survival (OS) [ Time Frame: 2 years ]
    PFS will be assessed based on the Response Assessment in Pediatric Neuro-Oncology (RAPNO) scale. Tumor related biomarkers will be compared before and after the BBBD procedure


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05615623


Contacts
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Contact: Nadir Alikacem +12146302000 nadira@insightec.com

Locations
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Canada, Ontario
Sunnybrook Research Institute Recruiting
Toronto, Ontario, Canada, M4N 3M5
Contact: James Rutka, MD    416-813-6425    james.rutka@sickkids.ca   
Contact: Maheleth Llinas    416-480-6100 ext 2476    maheleth.llinas@sunnybrook.ca   
Principal Investigator: Nir Lipsman, MD         
Principal Investigator: James Rutka, MD         
Sponsors and Collaborators
InSightec
More Information
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Responsible Party: InSightec
ClinicalTrials.gov Identifier: NCT05615623    
Other Study ID Numbers: BT016C
First Posted: November 14, 2022    Key Record Dates
Last Update Posted: January 6, 2023
Last Verified: January 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by InSightec:
DIPG
Focused Utrasound
Chemotherapy
Phase I trial
Pediatrics
Additional relevant MeSH terms:
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Brain Neoplasms
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Doxorubicin
Antibiotics, Antineoplastic
Antineoplastic Agents
Topoisomerase II Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action