Focused Ultrasound with neuro-navigator-controlled sonication for Diffuse Intrinsic Pontine Glioma

Phase-Based Estimates
1
Effectiveness
1
Safety
Columbia University Irving Medical Center / NewYork-Presbyterian Hospital, New York, NY
Diffuse Intrinsic Pontine Glioma+3 More
Focused Ultrasound with neuro-navigator-controlled sonication - Device
Eligibility
< 65
All Sexes
Eligible conditions
Diffuse Intrinsic Pontine Glioma

Study Summary

Non-Invasive Focused Ultrasound (FUS) With Oral Panobinostat in Children With Progressive Diffuse Midline Glioma (DMG)

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Eligible Conditions

  • Diffuse Intrinsic Pontine Glioma
  • Glioma
  • Diffuse Pontine and Thalamic Gliomas
  • Diffuse Midline Glioma, H3 K27M-Mutant

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Focused Ultrasound with neuro-navigator-controlled sonication will improve 1 primary outcome and 3 secondary outcomes in patients with Diffuse Intrinsic Pontine Glioma. Measurement will happen over the course of Up to 6 months after last FUS treatment..

Month 6
6-month Overall Survival (OS6)
6-month Progression Free Survival (PFS6)
Day 90
Blood brain barrier/Tumor imaging changes
Number of adverse events

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

Control
FUS using Oral Panobinostat

This trial requires 3 total participants across 2 different treatment groups

This trial involves 2 different treatments. Focused Ultrasound With Neuro-navigator-controlled Sonication is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

FUS using Oral PanobinostatAll patients enrolled in the study will be treated with oral Panobinostat after receiving Focused Ultrasound treatment (FUS) with microbubbles and neuro-navigator-controlled sonication.
ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 90 days after the end of the last fus treatment
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 90 days after the end of the last fus treatment for reporting.

Closest Location

Columbia University Irving Medical Center / NewYork-Presbyterian Hospital - New York, NY

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
The patient was diagnosed with a tumor involving the pons, thalami, and histologically confirmed to have the H3K27M mutation. show original
Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must have been off all prior anti-cancer therapy for a minimum of four weeks prior to enrollment. show original
not eligible for any other therapy (other than corticosteroids) will be ineligible for enrollment on this study Subjects with evidence of clinical and/or radiographic progression of Diffuse Midline Glioma, who are not eligible for any other therapy (other than corticosteroids), will not be enrolled on this study. show original
At least 21 days after the last dose of chemotherapy that is known to be cytotoxic or myelosuppressive. show original
before the patient may receive a transfusion of blood components show original
At least 21 days must have elapsed since the completion of interleukins, interferons, or cytokines. show original
Ages 4-21 is a range of ages. show original
Cancer agents that are not known to be myelosuppressive: You must wait at least 7 days from your last dose of cancer agent before using this product. show original
If you have received an antibody infusion, you must wait at least 21 days before receiving another one. show original
Patients who have recently undergone a stem cell infusion must wait at least 84 days before donating. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for diffuse intrinsic pontine glioma?

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In most patients with diffuse intrinsic pontine glioma, surgical resection of the tumor is indicated, but often the patient does not have a long-term prognostic advantage from this treatment. Radiochemotherapy is often used as first-line treatment. At the Mayo Clinic, this treatment has been associated with improvement in survival, but this result must be viewed with caution because most of the patients who had radiochemotherapy have had chemotherapy before and radiation therapy after.

Unverified Answer

How many people get diffuse intrinsic pontine glioma a year in the United States?

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This analysis is one of the largest cohorts of DAI patients reported in the literature. While overall survival is excellent, the median overall survival in newly diagnosed DAI is less than 6 months.

Unverified Answer

What is diffuse intrinsic pontine glioma?

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Symptoms can be variable. A mass effect on the brainstem should be ruled out. The extent of tumor should be assessed before treatment can be proposed. Early diagnosis of diffuse intrinsic pontine glioma is associated with good prognosis. Imaging at presentation (MRI) may be used for diagnosis. Patients should be considered at risk and be referred to a high-grade glioma/meningioma center. Diffuse intrinsic pontine glioma is a very aggressive disease with a long remission interval of years following initial treatment. Early diagnosis can impact the number of patient-related treatment and outcomes.

Unverified Answer

Can diffuse intrinsic pontine glioma be cured?

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Most children with DIPG who will die without signs of tumor recurrence have very good initial survival, particularly in cases with low-grade or asymptomatic disease. Long-term survival is poor in patients with high grade disease.

Unverified Answer

What causes diffuse intrinsic pontine glioma?

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The exact cause of diffuse intrinsic pontine glioma is unknown. Inherited genetic factors may play a significant role in developing diffuse intrinsic pontine glioma. Diffuse intrinsic Pontine Glioma (DIPG) may result from mutations in one of the genes of the Hedgehog pathway.

Unverified Answer

What are the signs of diffuse intrinsic pontine glioma?

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The most common symptoms of diffuse intrinsic pontine glioma are headache and visual impairment. A seizure diagnosis is the most common incidental finding in the presence of diffuse intrinsic pontine glioma. However, the seizure was a nonspecific indicator of diffuse intrinsic pontine glioma. Seizures can be caused by many conditions, and therefore a seizure diagnosis does not reliably indicate diffuse intrinsic pontine glioma. Tumor-associated ophthalmoplegia can happen in diffuse intrinsic pontine glioma, but the diagnosis can be supported by tumor cells in the orbital tissue. Tumor-associated ophthalmoplegia is a nonspecific presentation of diffuse intrinsic pontine glioma.

Unverified Answer

Is focused ultrasound with neuro-navigator-controlled sonication safe for people?

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This preliminary study suggests that focused ultrasound with SNC-NVS is safe for people with or without metal implants or implants in the brain tissue and does show a good feasibility and tolerability. More research is needed to confirm these findings and to extend the follow-up period.

Unverified Answer

Does diffuse intrinsic pontine glioma run in families?

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Recent findings suggests that DIPG may be under-diagnosed and, more than likely, some cases may simply go undiagnosed. The number of cases of DIPG currently diagnosed is likely greater than the number of cases which have been missed. There is a need to educate the public and healthcare professionals and to implement national initiatives to increase awareness of the disease and to support early diagnosis and appropriate treatment.

Unverified Answer

What are the chances of developing diffuse intrinsic pontine glioma?

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The risk for the development of DIPG over an average of 4 years is approximately 4/100,000 for women and less than 0.001 for men. These values can be used to counsel patients and their families about the risk.

Unverified Answer

What is the primary cause of diffuse intrinsic pontine glioma?

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It is a rare event for PGG to be caused by a primary brainstem glioma. Although the tumor was not surgically biopsy-proven, histologic findings, tumor localization, and clinical presentation strongly support PGG as a primary brainstem glioma. These lesions are typically treated with radiation therapy.

Unverified Answer

Have there been other clinical trials involving focused ultrasound with neuro-navigator-controlled sonication?

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This report indicates the efficacy and safety of NNC sonication with focussed ultrasound. The improvement of PVS in a clinical study, using NNC sonication in combination with FUS, warrants further study.

Unverified Answer

What does focused ultrasound with neuro-navigator-controlled sonication usually treat?

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This technique is useful to reduce the number of inlets to the tumor and to identify the best one. However, there were a lot of malpositioned inlets inside the tumor as well as other inelinities nearby which makes the ultrasound ineffective.

Unverified Answer
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