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Histone Deacetylase Inhibitor

Focused Ultrasound + Panobinostat for Brain Cancer

Phase 1
Waitlist Available
Led By Stergios Zacharoulis, MD
Research Sponsored by Columbia University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ages 4-21 years
Subjects with evidence of clinical and/or radiographic progression of Diffuse Midline Glioma
Must not have
Subjects with documented allergy to compounds of similar chemical or biologic composition to Panobinostat or gadolinium compounds
Subjects with an uncorrectable bleeding disorder
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 90 days after the end of the last fus treatment
Awards & highlights

Summary

This trial uses sound waves and tiny bubbles to temporarily open the brain's protective barrier, allowing more of the drug Panobinostat to reach brain tumors in children with a specific type of aggressive brain cancer. This method aims to improve treatment response and reduce side effects.

Who is the study for?
This trial is for children aged 4-21 with progressive diffuse midline glioma (DMG) who've recovered from prior cancer treatments. They must be able to undergo MRI scans, swallow capsules, and have a stable health condition as assessed by the investigator. Those with spinal DMGs, bleeding disorders, metal implants, or severe systemic diseases are excluded.
What is being tested?
The study tests if Focused Ultrasound (FUS) can safely open the blood-brain barrier in kids with DMG to enhance Panobinostat delivery to brain tumors. The non-invasive FUS will target up to three areas near the tumor while administering oral Panobinostat.
What are the potential side effects?
Potential side effects include reactions related to opening the blood-brain barrier such as temporary neurological changes or discomfort at sonication sites. Oral Panobinostat may cause digestive issues, fatigue, blood count changes and could affect heart rhythm.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 4 and 21 years old.
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My brain tumor is growing or spreading.
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My tumor in the brain stem or thalamus is confirmed by imaging or has a specific genetic change.
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I can swallow capsules.
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I am taking medication for seizures/epilepsy before my first surgery in the study.
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I am between 4 and 21 years old.
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I am mostly able to care for myself and carry out daily activities.
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I can have an MRI scan with a special dye.
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My liver and kidney functions are normal.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am allergic to Panobinostat or similar drugs, or to gadolinium.
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I have a bleeding disorder that cannot be corrected.
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I have signs of brain swelling or recent bleeding within a tumor.
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I have a brain blood vessel condition.
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I currently have an active infection.
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I have a bleeding disorder or am on blood thinners.
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My high blood pressure is not controlled by medication.
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My tumor is bleeding.
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I am using two effective birth control methods if my partner can get pregnant or if I can get pregnant.
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I have had a stroke or heart disease in the past.
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I have a spinal diffuse midline glioma.
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I cannot undergo general anesthesia due to a medical condition.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 90 days after the end of the last fus treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 90 days after the end of the last fus treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of adverse events
Secondary study objectives
6-month Overall Survival (OS6)
6-month Progression Free Survival (PFS6)
Blood brain barrier/Tumor imaging changes

Trial Design

1Treatment groups
Experimental Treatment
Group I: FUS using Oral PanobinostatExperimental Treatment2 Interventions
All patients enrolled in the study will be treated with oral Panobinostat after receiving Focused Ultrasound treatment (FUS) with microbubbles and neuro-navigator-controlled sonication.

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The primary treatment for Diffuse Intrinsic Pontine Glioma (DIPG) is radiation therapy, which works by targeting and damaging the DNA of cancer cells, thereby inhibiting their ability to replicate and grow. This treatment can temporarily alleviate symptoms and improve quality of life. Investigational therapies, such as Focused Ultrasound (FUS) with microbubbles and neuro-navigator-controlled sonication, aim to temporarily open the blood-brain barrier (BBB). This allows for enhanced delivery of chemotherapeutic agents like Panobinostat directly to the tumor site, potentially increasing the drug's efficacy while minimizing systemic side effects. These approaches are significant for DIPG patients as they offer hope for more effective treatment options in a disease that is notoriously difficult to treat due to its location and the protective nature of the BBB.

Find a Location

Who is running the clinical trial?

Focused Ultrasound FoundationOTHER
16 Previous Clinical Trials
371 Total Patients Enrolled
1 Trials studying Diffuse Intrinsic Pontine Glioma
10 Patients Enrolled for Diffuse Intrinsic Pontine Glioma
Columbia UniversityLead Sponsor
1,480 Previous Clinical Trials
2,621,855 Total Patients Enrolled
Cheng-Chia (Fred) WuLead Sponsor
1 Previous Clinical Trials
10 Total Patients Enrolled
1 Trials studying Diffuse Intrinsic Pontine Glioma
10 Patients Enrolled for Diffuse Intrinsic Pontine Glioma

Media Library

Panobinostat (Histone Deacetylase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04804709 — Phase 1
Diffuse Intrinsic Pontine Glioma Research Study Groups: FUS using Oral Panobinostat
Diffuse Intrinsic Pontine Glioma Clinical Trial 2023: Panobinostat Highlights & Side Effects. Trial Name: NCT04804709 — Phase 1
Panobinostat (Histone Deacetylase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04804709 — Phase 1
~1 spots leftby Oct 2025