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Trial Title:
Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric DIPG
NCT ID:
NCT05630209
Condition:
Brain Tumor
Conditions: Official terms:
Brain Neoplasms
Doxorubicin
Conditions: Keywords:
DIPG
Focused Ultrasound
Chemotherapy
Phase I trial
Pediatrics
Study type:
Interventional
Study phase:
Phase 1/Phase 2
Overall status:
Recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Device Feasibility
Masking:
None (Open Label)
Intervention:
Intervention type:
Device
Intervention name:
Exablate
Description:
Blood Brain Barrier Disruption (BBBD) via Exablate Type 2 system with microbubble
resonators on the day of Doxorubicin infusion to treat DIPG brain tumors
Arm group label:
Blood Brain Barrier Disruption (BBBD)
Other name:
Exablate BBBD
Intervention type:
Drug
Intervention name:
Doxorubicin
Description:
Doxorubicin infusion
Arm group label:
Blood Brain Barrier Disruption (BBBD)
Other name:
Adriamycin
Other name:
Rubex
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
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.
Criteria for eligibility:
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
Gender:
All
Minimum age:
5 Years
Maximum age:
21 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Children's National Medical Center
Address:
City:
Washington
Zip:
20010
Country:
United States
Status:
Recruiting
Contact:
Last name:
Elizabeth Paronett
Phone:
202-476-5551
Email:
eparonett@childrensnational.org
Investigator:
Last name:
Roger Packer
Email:
Principal Investigator
Investigator:
Last name:
Robert Keating
Email:
Sub-Investigator
Facility:
Name:
Nicklaus Children's Hospital
Address:
City:
Miami
Zip:
33155
Country:
United States
Status:
Recruiting
Contact:
Last name:
Toba Niazi, MD
Phone:
305-662-8386
Email:
Toba.niazi@nicklaushealth.org
Contact backup:
Last name:
Ana Diaz, ARNP
Email:
DIPG.ClinicalTrial@nicklaushealth.org
Investigator:
Last name:
Toba Niazi, MD
Email:
Principal Investigator
Start date:
January 4, 2023
Completion date:
January 2026
Lead sponsor:
Agency:
InSightec
Agency class:
Industry
Source:
InSightec
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05630209