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

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ClinicalTrials.gov Identifier: NCT05630209
Recruitment Status : Recruiting
First Posted : November 29, 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 Type2.0/2.1 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 up to three sites in the United States. 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 : 10 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)
Estimated Study Start Date : February 2023
Estimated Primary Completion Date : January 2025
Estimated Study Completion Date : January 2026

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. Progression Free Survival (PFS) [ Time Frame: 2 years ]
    PFS will be assessed based on the Response Assessment in Pediatric Neuro-Oncology (RAPNO) scale.


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age between 5 and 21 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
  • 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
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): NCT05630209


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

Locations
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United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Elizabeth Paronett    202-476-5551    eparonett@childrensnational.org   
Principal Investigator: Roger Packer         
Sub-Investigator: Robert Keating         
Sponsors and Collaborators
InSightec
More Information
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Responsible Party: InSightec
ClinicalTrials.gov Identifier: NCT05630209    
Other Study ID Numbers: BT016
First Posted: November 29, 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: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Keywords provided by InSightec:
DIPG
Focused Ultrasound
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