← Back to Search

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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 90 days after the end of the last fus treatment
Awards & highlights

Study Summary

This trial is being done to see if it is safe to open the blood brain barrier in children with brain cancer using focused ultrasound technology, and to administer the oral cancer drug Panobinostat.

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.Check my eligibility
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.See study design
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
Select...
I am between 4 and 21 years old.
Select...
My brain tumor is growing or spreading.
Select...
My tumor in the brain stem or thalamus is confirmed by imaging or has a specific genetic change.
Select...
I am healthy enough for FUS, MRI, and any required anesthesia, as per my doctor.
Select...
I can swallow capsules.
Select...
I am taking medication for seizures/epilepsy before my first surgery in the study.
Select...
I have recovered from side effects of my previous cancer treatments.
Select...
I am between 4 and 21 years old.
Select...
I am mostly able to care for myself and carry out daily activities.
Select...
I can have an MRI scan with a special dye.
Select...
I have been on a stable or decreasing dose of steroids and anti-seizure medication for at least 1 week.
Select...
My liver and kidney functions are normal.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of adverse events
Secondary outcome measures
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.

Find a Location

Who is running the clinical trial?

Columbia UniversityLead Sponsor
1,424 Previous Clinical Trials
2,473,024 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
Focused Ultrasound FoundationOTHER
14 Previous Clinical Trials
380 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

Frequently Asked Questions

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

Does this clinical trial permit participation from individuals who are senior citizens?

"Participants between the ages of 4 and 21 are eligible to join this clinical trial."

Answered by AI

Does this experiment involve any groundbreaking research?

"To date, 14 independent clinical trials for Focused ultrasound with neuro-navigator-controlled sonication have been launched in 95 cities spread across 32 nations. The pioneer study was bankrolled by Secura Bio, Inc., and featured 18 participants; it ended its Phase 1 drug approval stage in 2010. Since then, 72 more studies have taken place."

Answered by AI

Could you discuss any previous research on using neuro-navigator-controlled sonication with Focused Ultrasound?

"Currently, 14 trials for Focused ultrasound with neuro-navigator-controlled sonication are running. Not one of those is in Phase 3 yet. Although the main hub for these studies appears to be New york City, 201 total locations across America are participating in this research effort."

Answered by AI

Do I meet the prerequisites to join this investigation?

"In order to be considered for this clinical trial, participants must have a diagnosis of diffuse intrinsic pontine glioma and belong within the age group 4-21. Currently 3 individuals are required to take part in the research program."

Answered by AI

What safety protocols have been established for Focused Ultrasound with neuro-navigator-controlled sonication?

"As this is an early stage Phase 1 trial, there are only minimal data supporting the safety and efficacy of Focused ultrasound with neuro-navigator-controlled sonication; thus it received a score of 1."

Answered by AI

Are there any opportunities remaining to participate in this research endeavor?

"Clinicaltrials.gov confirms that this medical research project is no longer taking on participants; it was initially listed on July 28, 2021 and its last update happened February 3rd 2022. Even though participation in the study has now closed, there are 378 alternative trials presently searching for volunteers."

Answered by AI

How many participants are eligible to join this research endeavor?

"No longer in the process of searching for applicants, this medical study was initially advertised on July 28th 2021 and edited as recently as February 3rd 2022. For those looking for alternative clinical trials, there are now 364 studies seeking diffuse intrinsic pontine glioma patients and 14 research projects recruiting participants to test Focused ultrasound with neuro-navigator-controlled sonication."

Answered by AI

Who else is applying?

How old are they?
< 18
What site did they apply to?
Columbia University Irving Medical Center / NewYork-Presbyterian Hospital
What portion of applicants met pre-screening criteria?
Met criteria
~1 spots leftby Mar 2025