Optune for Children With High-Grade Glioma or Ependymoma, and Optune With Radiation Therapy for Children With DIPG
Feasibility Trial of Optune for Children With Recurrent or Progressive Supratentorial High-Grade Glioma or Ependymoma, and Feasibility and Efficacy Trial of Optune in Conjunction With Radiation Therapy for Children With Newly Diagnosed DIPG
3 other identifiers
interventional
80
1 country
11
Brief Summary
This is a multicenter trial of the Optune device to examine the feasibility and to describe the device-related toxicity in children with supratentorial high grade glioma (HGG) or ependymoma (Stratum 1) and to examine the feasibility and efficacy of concurrent Optune and standard focal radiation therapy (RT) in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG) (Stratum 2).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for not_applicable
Started Apr 2017
Longer than P75 for not_applicable
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 17, 2017
CompletedFirst Posted
Study publicly available on registry
January 27, 2017
CompletedStudy Start
First participant enrolled
April 4, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 22, 2031
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 21, 2031
March 5, 2026
August 1, 2025
14.1 years
January 17, 2017
March 3, 2026
Conditions
Outcome Measures
Primary Outcomes (5)
The feasibility of treatment with the Optune device in pediatric patients with recurrent/refractory/progressive supratentorial malignant glioma and ependymoma.
Stratum 1: Device Usage Compliance will be reported to assess the feasibility of the device treatment.
days 8 through 35 of cycle 1
The Optune device treatment-related toxicities in children with recurrent/refractory/progressive supratentorial malignant glioma and ependymoma.
Stratum 1: Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0.
2 years
The type and frequency of Device Limiting Toxicities (DLTs) associated with the Optune treatment given concurrently with RT in children and adolescents with newly diagnosed DIPG.
Stratum 2:The number of participants with DLTs per protocol Section 5.4.
5 years
The feasibility of concurrent Optune therapy and RT in children and adolescents with newly diagnosed DIPG.
Stratum 2: Device Usage Compliance will be reported to assess the feasibility of the device treatment when administered concurrent with RT. The device treatment will be considered feasible for a patient who use the device for ≥18 hours/day for at least 40 of 49 days (23 of 28 days if the second dose de-escalation is utilized for phase II) of the feasibility period of Cycle 1.
days 8 through 56 of cycle 1 (days 8 through 35 of cycle 1 if the second dose de-escalation is utilized for phase II)
The overall survival
Stratum 2: OS is defined as time from enrollment to death due to any cause.
3 years
Secondary Outcomes (10)
The Response Rate
2 years
The Event-Free Survival
2 years
The association of anti-tumor activity with compliance in Optune device use within the context of a small feasibility study.
2 years
The association between the Optune device usage and the health-related quality of life of children and families undergoing this therapy.
2 years
The association of apparent diffusion coefficient (ADC) values within the tumor and correlate with response to Optune treatment and EFS.
2 years
- +5 more secondary outcomes
Study Arms (2)
Treatment (Optune System)
EXPERIMENTALPatients must have a histologically confirmed diagnosis of supratentorial high-grade glioma or supratentorial ependymoma that is recurrent, progressive or refractory. All patients will use the study device Optune System (Tumor Treating Fields, TTFields).
Treatment (Concurrent Optune/focal radiation therapy followed by Optune-only therapy)
EXPERIMENTALPatients must have newly diagnosed DIPG (a typical DIPG on MR imaging, defined as a tumor with a pontine epicenter and diffuse involvement of more than 2/3 of the pons, without histologic confirmation; atypical DIPG undergoing a biopsy and the tumor is a diffuse glioma WHO Grade II-IV with OR without H3 K27M mutation; or pontine lesions that do not meet these MR imaging criteria with histologically confirmed diffuse glioma WHO Grade II-IV with H3 K27M- mutation). This arm will consist of two parts: a phase I component to evaluate the safety and tolerability of concurrent Optune and RT, and a phase II component to evaluate the feasibility of concurrent Optune and RT and the efficacy associated with this approach compared to historical controls.
Interventions
The Optune, a.k.a. Tumor Treating Fields (TTFields), will be worn for a minimum of 18 hours a day, with a recommendation of 22 hours/day for at least 23 days in a 28-day cycle. Cycle 1 includes 7 days training period, followed by 28 days treatment (total 35 days). The patients will receive multiple 28-day cycles of continuous Optune treatment. In the absence of treatment related serious adverse events or disease progression, Optune will continue up to 26 cycles.
In Stratum II patients will be treated with standard of care focal RT concurrently with Optune followed by Optune treatment. This study specifies a 1 cm clinical target volume margin.The duration of the first cycle of therapy will be from Day 1 of RT to 14 days after completion of RT (approximately 8 weeks total). Subsequent cycles of therapy will be 28 days in duration. The Optune device will be worn for a minimum of 18 hours/day, with a recommendation of 22 hours/day. For the phase I part, the initial treatment will involve RT delivery with Optune arrays remaining in place but turned off. If this is not tolerated, two treatment de-escalation levels are planned, Specifically, Level -1: remove the Optune arrays during RT delivery and reapply daily after RT; Level -2: initiate Optune therapy after completion of RT. The established safe treatment approach from phase I will be investigated for feasibility and efficacy in phase II. Treatment with Optune may continue for up to 5 years.
Eligibility Criteria
You may qualify if:
- Age: at the time of enrollment Stratum 1: ≥ 5 years but ≤ 21 Stratum 2: ≥ 3 years but ≤ 21
- Diagnosis:
- Stratum 1: Patients must have a histologically confirmed diagnosis of supratentorial high-grade glioma or supratentorial ependymoma that is recurrent, progressive or refractory.
- Stratum 2: Patients with newly diagnosed DIPG
- Patients with a typical DIPG on MR imaging, defined as a tumor with a pontine epicenter and diffuse involvement of more than 2/3 of the pons, are eligible without histologic confirmation.
- Note: Patients with typical DIPG who undergo a biopsy are eligible provided the tumor is a diffuse glioma WHO Grade II-IV with OR without H3 K27M mutation.
- Patients with pontine lesions that do not meet these MR imaging criteria will be eligible if there is histologic confirmation diffuse glioma WHO Grade II-IV with H3 K27M-mutation.
- Disease Status: Patients must have bi-dimensionally measurable disease, defined as at least one lesion that can be accurately measured in at least two planes
- Stratum 1:
- This disease must be located primarily in the supratentorial region
- Patients with significant disease that is metastatic outside of the supratentorial region are ineligible
- Stratum 2:
- This disease must be located primarily in the pons
- Prior Therapy:
- Stratum 1: Patients must have recovered from the acute treatment related toxicities (defined as ≤ grade 1) of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
- +35 more criteria
You may not qualify if:
- Systemic Illness: Patients with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the opinion of the investigator would compromise the patient's ability to tolerate protocol therapy, put them at additional risk for toxicity or would interfere with the study procedures or results.
- Other Malignancy: Patients with a history of any other malignancy.
- Concurrent Therapy: Patients who are receiving any other anticancer or investigational drug therapy are not eligible.
- Inability to Participate: Patients who in the opinion of the investigator are unwilling or unable to return for required follow-up visits or obtain follow-up studies required to assess toxicity to therapy or to adhere to device usage plan, other study procedures, and study restrictions.
- Stratum 1 Tumor Location: Patients with primarily infra-tentorial or spinal cord tumor are not eligible.
- Tumor Dissemination: Patients for who clinical suspicion is present of metastatic disease in the CSF or Spine must have MRI of Spine and CSF obtained (Lumbar puncture or through ommaya, EVD or Shunt) with negative cytology. Patients with CSF that is positive for tumor cells or metastatic disease found on MRI are ineligible.
- Skull Defects: Patients with major skull defects (such as missing bone without replacement) are not eligible.
- Neurological Disorder: Patients with active implanted electronic devices in the brain or spinal cord such as programmable VP shunts, deep brain stimulators, vagus nerve stimulators, are not eligible.
- Cardiac Disorder: Patients with pacemaker, defibrillator, or documented significant arrhythmia, are not allowed.
- Intracranial Objects: Patients with foreign body intracranially, such as bullet fragments, are not allowed, with the exception of VP-shunts (non-programmable) and Ommaya catheters.
- Allergy: Patients with history of hypersensitivity to conductive hydrogel are not eligible.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- NovoCure Ltd.collaborator
- Children's Oncology Grouplead
- National Cancer Institute (NCI)collaborator
- American Lebanese Syrian Associated Charitiescollaborator
- Pediatric Brain Tumor Consortiumcollaborator
Study Sites (11)
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Children's Hospital Los Angeles
Los Angeles, California, 90026, United States
Lucile Packard Children's Hospital at Stanford University Medical Center
Palo Alto, California, 94304, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010-2970, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Stewart Goldman, MD
Phoenix Children's Hospital
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Masking Details
- Open-label single-arm study within each stratum
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 17, 2017
First Posted
January 27, 2017
Study Start
April 4, 2017
Primary Completion (Estimated)
May 22, 2031
Study Completion (Estimated)
July 21, 2031
Last Updated
March 5, 2026
Record last verified: 2025-08
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, ICF, CSR
The Pediatric Brain Tumor Consortium (PBTC) ensures that high dimensional molecular data generated from PBTC samples will be submitted to the NIH Database of Genotypes and Phenotypes (dbGaP) or other NIH-designated data repository. PBTC is required to make data available to other investigators for use in research projects. An investigator requesting data from published PBTC studies will submit a proposal describing the studies from which data are requested, the requested data, and a list of investigators in the project and their affiliated institutions, along with the investigator's CV to the PBTC Steering Committee for review. Once approved, the requesting investigator will be asked to complete a Data Transfer Agreement before the release of deidentified data. Requests for data are typically only considered once the primary study manuscript has been published though exceptions may be made in the event of long delays in the primary study data publication.