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Histone Deacetylase Inhibitor

Panobinostat for Brain Tumor (PBTC-047 Trial)

Phase 1
Waitlist Available
Led By Michelle Monje, MD, Phd
Research Sponsored by Pediatric Brain Tumor Consortium
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with Thalamic Diffuse Midline Glioma with H3K27M mutation confirmation
Patients aged ≥ 2 but < 22 years at enrollment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up urine samples were collected for cell-free dna based assay at course 1 day 1 (c1d1), c2d1, c4d1, and c6d1.
Awards & highlights

PBTC-047 Trial Summary

This trial is testing panobinostat to treat DIPG, a type of brain tumor. The trial will test different doses of the drug to see what the side effects are and what the best dose is. The trial is enrolling patients who have DIPG that has not yet gotten worse.

Who is the study for?
This trial is for children and young adults aged 2 to 21 with a specific brain tumor called Diffuse Intrinsic Pontine Glioma (DIPG) or H3K27M+ Thalamic Diffuse Malignant Glioma (DMG). Participants must have tumors that are growing or worsening, but currently only those without progression can join. They need a certain body surface area depending on the dose level.Check my eligibility
What is being tested?
The trial is testing Panobinostat (LBH589), which may halt tumor growth by inhibiting enzymes needed for cell division. There are two parts: one for patients whose DIPG has worsened, and another for those with stable disease; however, only the latter group is being enrolled now.See study design
What are the potential side effects?
Panobinostat's side effects in children with these brain tumors aren't fully known yet—that's part of what this study aims to find out. Generally, it could include fatigue, nausea, diarrhea, blood count changes, and other enzyme-related effects.

PBTC-047 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My brain tumor is a Thalamic Diffuse Midline Glioma with an H3K27M mutation.
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I am between 2 and 21 years old.
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I am a woman who can have children and my pregnancy test is negative.
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I have had targeted radiation therapy before joining.
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I can do most of my daily activities by myself.
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I am eligible for radiation therapy.
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My treatment dose is based on my body size.
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My diagnosis of DIPG is based on scans without a biopsy.
Select...
My brain tumor is confirmed as a high-grade glioma.
Select...
My brain tumor is growing or I have a specific type of brain tumor (H3K27M+).

PBTC-047 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from date on treatment until date of pd or death due to any cause or date of last follow-up
This trial's timeline: 3 weeks for screening, Varies for treatment, and from date on treatment until date of pd or death due to any cause or date of last follow-up for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Area under the Curve (AUC)
Clearance (CL/F)
Elimination Rate (Kel)
+5 more
Secondary outcome measures
Overall Survival (OS) in Stratum 1
Overall Survival (OS) in Stratum 2
Percentage of Patients with H3F3A K27M Mutation Detected in Blood Samples
+5 more

Side effects data

From 2016 Phase 2 trial • 13 Patients • NCT01013597
23%
Death, NOS
8%
Colitis, infectious
8%
Neutrophils/granulocytes (ANC/AGC)
8%
Platelets
8%
Hemorrhage/bleeding
8%
CNS cerebrovascular ischemia
8%
Other
8%
Pleural effusion, non-malignant
8%
Thrombosis/thrombus/embolism
100%
80%
60%
40%
20%
0%
Study treatment Arm
LBH589

PBTC-047 Trial Design

2Treatment groups
Experimental Treatment
Group I: Treatment (STRATUM 2)Experimental Treatment1 Intervention
Patients with non-progressed DIPG or H3K27M+ Thalamic DMG will be enrolled. All patients will take the study drug panobinostat (LBH589).
Group II: Treatment (STRATUM 1)Experimental Treatment1 Intervention
Patients with recurrent/progressive DIPG will be enrolled at the time of progression. All patients will take the study drug panobinostat (LBH589).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
LBH589
2010
Completed Phase 2
~670

Find a Location

Who is running the clinical trial?

Pediatric Brain Tumor ConsortiumLead Sponsor
37 Previous Clinical Trials
1,529 Total Patients Enrolled
Michelle Monje, MD, PhdPrincipal InvestigatorStanford University

Media Library

Panobinostat (Histone Deacetylase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT02717455 — Phase 1
Brain Tumor Research Study Groups: Treatment (STRATUM 1), Treatment (STRATUM 2)
Brain Tumor Clinical Trial 2023: Panobinostat Highlights & Side Effects. Trial Name: NCT02717455 — Phase 1
Panobinostat (Histone Deacetylase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02717455 — Phase 1

Frequently Asked Questions

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

Are there any vacancies in this clinical trial that a patient could potentially fill?

"At present, it appears that this medical trial is not currently seeking applicants. It was first posted on June 28th 2016 and last updated August 8th 2022. Nevertheless, there are 362 trials actively recruiting patients with glioma and another 14 studies admitting participants for LBH589 research."

Answered by AI

What is the current participant count for this research study?

"Unfortunately, this clinical trial is no longer enrolling patients. While it was initially posted on the 28th of June 2016 and last updated 8th August 2022, there are currently 362 trials related to glioma actively searching for participants and 14 studies involving LBH589 that require volunteers."

Answered by AI

Is geriatric participation permissible in this experiment?

"According to the entry requirements for this experiment, participants must be within the age range of 2 and 21. Furthermore, there are 146 trials available for minors under 18 years old and 248 studies that cater to individuals over 65 years of age."

Answered by AI

Am I a qualifying participant for this research initiative?

"Those that wish to be included in this clinical trial must have glioma and fall within the age range of 2-21. This research is currently attempting to recruit around 53 individuals."

Answered by AI

Has LBH589 been tested in other medical experiments?

"Presently, there are 14 live clinical trials researching LBH589 and none of them have entered Phase 3. The bulk of these studies occur in New york City but there is also access to research at 201 other sites."

Answered by AI

How many separate places is this research endeavor being coordinated?

"Patients can join this study in Cincinnati Children's Hospital Medical Center, located in Ohio; Children's Hospital of Pittsburgh (Pennsylvania); and the Washington-based Children's National Medical Centre. Additionally, there are 10 other participating sites."

Answered by AI

Is this research the pioneer of its kind?

"Currently, there are 14 active trials for LBH589 spanning 95 municipalities and 32 nations. Secura Bio, Inc. began this journey in 2010 with its Phase 1 study which encompassed 18 patients - since then 72 separate studies have concluded their investigations."

Answered by AI

What adverse effects have been reported in connection with LBH589 consumption?

"All the extant data suggests LBH589 is safe to use, so our team assigned it a score of 1. This rating aligns with its status as a Phase 1 clinical trial, where there are only preliminary indications that the drug may be effective and have an acceptable level of risk."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma
2016. "Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02717455.
~6 spots leftby Apr 2025
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