for people ages 2-21 (full criteria)
at San Francisco, California and other locations
study started
estimated completion
Principal Investigator
by Sabine Mueller
Photo of Sabine Mueller
Sabine Mueller



This phase I/II trial studies the side effects of panobinostat nanoparticle formulation MTX110 (MTX110) in treating participants with newly-diagnosed diffuse intrinsic pontine glioma. Panobinostat nanoparticle formulation MTX110 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Official Title

An Open Label Single Arm Phase I/II Study of MTX110 Delivered by Convection-enhanced Delivery (CED) in Patients With Diffuse Intrinsic Pontine Glioma (DIPG) Previously Treated With External Beam Radiation Therapy


PRIMARY OBJECTIVES: I. To determine the safety and tolerability of repeated administration of MTX110 co-infused with gadoteridol given by intratumoral convection enhanced delivery in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG). SECONDARY OBJECTIVES: I. To determine the clinical efficacy of repeated administration of MTX110 given by intratumoral convection-enhanced delivery (CED) in children with newly diagnosed DIPG in the confines of a phase I and early efficacy study. OUTLINE: This is a phase I, dose-escalation study followed by a phase II study. Participants receive panobinostat nanoparticle formulation MTX110 intratumorally (IT) by CED infusion on day 1 or days 1 and 2 as determined by dose level. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. After completion of study treatment participants are followed up at 30 days and then every 2 months.


Diffuse Intrinsic Pontine Glioma Glioma Panobinostat Panobinostat Nanoparticle Formulation MTX110 Convection-Enhanced Delivery (CED)


You can join if…

Open to people ages 2-21

  • Patients with newly diagnosed DIPG by magnetic resonance imaging (MRI); defined as patients with a pontine location and diffuse involvement of at least 2/3 of the pons are eligible without histologic diagnosis. For lesions with typical imaging features, biopsy is neither encouraged nor required for eligibility. Tumors that are biopsied will be eligible if proven to be supportive of the diagnosis of a DIPG. Consensus of diagnosis by the study team must be met.
  • Patients who have completed focal radiotherapy within 14 weeks from time of enrollment are eligible.
  • Treatment must begin at a minimum of 4 weeks after, but no later than 14 weeks after, the date of completion of focal radiotherapy.
  • Prior chemotherapy: Patients should be at least 30 days from last chemotherapy dose prior to start of CED infusion, with exception of antibody half-lives. For antibody therapies, at least 3 half-lives of the antibody after last dose of monoclonal antibody should have passed prior to CED infusion. Patients less than 30 days from last chemotherapy dose should be discussed with the study chair(s).
  • Prior radiation: Patients must have received prior treatment with focal radiotherapy as part of initial treatment for DIPG and had their last dose at least 4 weeks prior to and no later than 14 weeks from the first CED treatment. Standard focal radiation therapy will include 54 to 60 Gy by external beam radiotherapy to the brainstem.
  • Age ≥ 2 years of age to 21 years. Patients younger than 3 years of age may be enrolled on study at the discretion of the Study Chair(s) if supporting evidence that brainstem lesion represents a brainstem glioma.
  • Karnofsky Performance Score ≥ 50 for patients > 16 years of age and Lansky Performance Score ≥ 50 for patients ≤ 16 years of age. Patients who are unable to walk because of paralysis, but who are able to mobilize using a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  • Life expectancy of greater than 12 weeks measured from the date of completion of radiotherapy.
  • Corticosteroids: Patients who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration.
  • Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3.

  • Hemoglobin ≥ 8g/dl.
  • Platelet count ≥ 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).

  • Normal coagulation defined as normal International Normalized Ratio (INR) or per institutional guidelines.
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 70 milliliters (mL)/minute (min)/1.73 m2.

  • A serum creatinine (mg/dL) based on age/gender as follows:
  • Age: 2 to < 6 years; Male: 0.8; Female: 0.8
  • Age: 6 to < 10 years; Male: 1; Female: 1
  • Age: 10 to < 13 years; Male: 1.2; Female: 1.2
  • Age: 13 to < 16 years; Male:1.5; Female: 1.4
  • Age ≥ 16 years; Male: 1.7; Female: 1.4
  • Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age.
  • Serum glutamate pyruvate transaminase (SGPT) [alanine aminotransferase (ALT)] ≤ 110 U/L.
  • Serum albumin ≥ 2 g/dL.
  • Patients with seizure disorder may be enrolled if on non-enzyme inducing anticonvulsants and well controlled.
  • The effects of MTX110 on the developing human fetus are unknown. For this reason women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and 4 months after completion of MTX110 injection administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
  • Able to understand, and willing to sign, a written informed consent document.
  • Patients who are unable to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.

You CAN'T join if...

  • Patients who had clinical and/or radiographic (MRI) progression of tumor following external beam radiation therapy.
  • Patients with metastatic disease, including leptomeningeal or subarachnoid disseminated disease.
  • Patients with tumor morphology that predicts poor coverage of the majority of the tumor including bilateral thalamic involvement, or cysts that represent > 50% of cross-sectional areas of the pons. These subjects should be discussed with the study chairs.
  • Patients who are receiving any other investigational agents or other tumor-directed therapy.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to MTX110 or gadolinium.
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Female patients of childbearing potential must not be pregnant or breast-feeding. Female patients of childbearing potential must have a negative serum or urine pregnancy test within 14 days of registration.
  • Patients with MRI or clinical evidence of uncontrolled tumor mass effect are excluded; the assessment of mass effect should be made by the study chairs and study neurosurgeons prior to any planned CED treatment.
  • Untreated symptomatic hydrocephalus determined by treating physician.
  • Patients with evidence of intra-tumoral hemorrhage > 5 mm maximal diameter. These subjects should be discussed with the study chair.
  • Subjects with prolonged corrected QT (QTc) (> 450 msec) will be excluded from the study.


  • University of California, San Francisco
    San Francisco California 94158 United States
  • Memorial Sloan Kettering Cancer Center
    New York New York 10065 United States

Lead Scientist at UCSF


in progress, not accepting new patients
Start Date
Completion Date
Sabine Mueller, MD, PhD
Phase 1/2
Study Type
Last Updated