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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
Must not have
Patients receiving other anticancer or investigational drug therapy
Patients required to receive medication prolonging QTc interval
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from date on treatment until date of death due to any cause or date of last follow-up
Awards & highlights

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.
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.
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.

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.
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My brain tumor is confirmed as a high-grade glioma.
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My brain tumor is growing or I have a specific type of brain tumor (H3K27M+).

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am not on any other cancer or experimental drugs.
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I need to take medication that can affect my heart's rhythm.
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I am willing and able to follow the study's procedures.
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I have a digestive issue that affects medication absorption.
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I have severe diarrhea.
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I have received more than 60 Gy of radiation to the pons area.
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I have received specific treatments for a brain tumor before.
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I have had a bone marrow transplant in the past.
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My neurological condition has worsened significantly recently.
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I have had cancer before.
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My body does not respond to blood or platelet transfusions.

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.
This trial's timeline: 3 weeks for screening, Varies for treatment, and urine samples were collected for cell-free dna based assay at course 1 day 1 (c1d1), c2d1, c4d1, and c6d1. 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

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
~720

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)NIH
13,842 Previous Clinical Trials
41,002,947 Total Patients Enrolled
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
~6 spots leftby Sep 2025
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