Panobinostat for Brain Tumor (PBTC-047 Trial)

Palo Alto (17 mi)

Overseen byMichelle Monje, MD, Phd

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Pediatric Brain Tumor Consortium

No Placebo Group

Approved in 2 jurisdictions

Trial Summary

What is the purpose of this trial?This phase I trial studies the side effects and best dose of panobinostat in treating younger patients with diffuse intrinsic pontine glioma (DIPG). Panobinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Stratum 1 treats patients with DIPG that has returned or gotten worse (progressed). Stratum 2 treats patients with DIPG or H3K27+Thalamic Diffuse Malignant Glioma (DMG) that has not yet gotten worse.

Eligibility Criteria

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.

Inclusion Criteria

My brain tumor is a Thalamic Diffuse Midline Glioma with an H3K27M mutation.

I am between 2 and 21 years old.

I am a woman who can have children and my pregnancy test is negative.

I have had targeted radiation therapy before joining.

I can do most of my daily activities by myself.

I am eligible for radiation therapy.

My treatment dose is based on my body size.

My diagnosis of DIPG is based on scans without a biopsy.

My brain tumor is confirmed as a high-grade glioma.

My brain tumor is growing or I have a specific type of brain tumor (H3K27M+).

Exclusion Criteria

I am not on any other cancer or experimental drugs.

I need to take medication that can affect my heart's rhythm.

I am willing and able to follow the study's procedures.

I have a digestive issue that affects medication absorption.

I have severe diarrhea.

I have received more than 60 Gy of radiation to the pons area.

I have received specific treatments for a brain tumor before.

I have had a bone marrow transplant in the past.

My neurological condition has worsened significantly recently.

I have had cancer before.

My body does not respond to blood or platelet transfusions.

Treatment Details

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.

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).

Panobinostat is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Farydak for: