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


Trial Requirements and Treatment

This is an open-label, ascending dose, single arm phase I/II study of MTX110 delivered by CED in patients with DIPG following standard of care focal radiotherapy. We will start with a single dose drug concentration of 30 μM using a total volume of 3mL and administer MTX110 on day 1 only; we will then first dose escalate by the number of days MTX110 is given: 30 μM concentration, 3 ml total volume, administration will occur on day 1 and 2. We then will start to dose escalate the total volume that is being administered on days 1 and 2. The accelerated dose escalation design will allow intra-patient dose escalation. The concentration of gadoteridol (ProHance) will be 1 mM which is the same concentration that is being used for other studies; both agents will be combined and co-infused via the same catheters. A maximum of 2 catheters will be placed.

Rationale for Study

The overall median survival of children with DIPG is approximately 9 months, and remains unchanged despite decades of clinical trial research. The only standard of care is focal radiotherapy but essentially all children die of this disease. New therapeutic strategies are urgently needed. One of the potential reasons for failure of treatment is the blood-brain and blood-tumor barriers, which exclude potentially effective therapeutic agents. Direct delivery by convection-enhanced techniques can overcome this barrier and ensure adequate drug exposure to tumor cells. MTX110 is a soluble from of panobinostat. Panobinostat has been shown in pre-clinical models to be effective at slowing tumor growth in patient-derived brainstem xenografts, and these findings were seen both among carriers of histone mutations and wildtype histone models. The hypothesis of this study is that repeated direct delivery via convection-enhanced delivery (CED) of MTX110 will increase progression-free and overall survival in children with newly diagnosed DIPG following standard of care radiotherapy. This trial will assess the safety and preliminary efficacy of this strategy.

Primary Objectives

To determine the safety and tolerability of repeated administration of MTX110 co-infused with gadoteridol given by intratumoral CED in children with newly diagnosed DIPG.

Secondary Objectives 

To determine the clinical efficacy of repeated administration of MTX110 given by intratumoral CED in children with newly diagnosed DIPG in the confines of a phase I/II study.


Funding is provided by Cannonball Kids’ CancerThe Cure Starts Now, and Midatech Pharma Inc.

How to Enroll

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