NCT04341311

Phase I Study of Marizomib + Panobinostat for Children With DIPG

Phase 1 Trial of Marizomib Alone and in Combination With Panobinostat for Children With Diffuse Intrinsic Pontine Glioma

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Dana-Farber Cancer Institute · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

This research study is evaluating the safety, tolerability and preliminary efficacy of the drugs marizomib and panobinostat in pediatric patients with diffuse intrinsic pontine glioma (DIPG). The names of the study drugs involved in this study are: * Marizomib * Panobinostat

Detailed description

This research study involves chemotherapy as a possible treatment for pediatric patients with Diffuse intrinsic pontine glioma (DIPG) * The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits. * The names of the study drugs involved in this study are: * Marizomib * Panobinostat This study consists of 2 parts: * Part A : The investigators are looking at the highest dose (up to a targeted maximum dose) of the study drug Marizomib that can be administered safely without severe or unmanageable side effects in participants that have DIPG, not everyone who participates in this research study will receive the same dose of the study intervention. The dose given will depend on the number of participants who have been enrolled prior and how well the dose was tolerated. * Part B: Participants who tolerate Marizomib alone will be treated with Marizomib and panobinostat, but at lower dose of marizomib than the dose of marizomib when given alone. Participants are expected to be on study treatment for up to 2 years followed for up to 5 years. It is expected that up to 45 people will take part in this research study. This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the appropriate dose of the investigational drug to use for further studies. "Investigational" means that the drug is being studied. * Marizomib has not been approved by the U.S. Food and Drug Administration (FDA) as a treatment for any disease. \-- This is the first time Marizomib will be given to children. * Panobinostat has not approved by the U.S. Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma but it has been approved for other uses.

Conditions

• Diffuse Intrinsic Pontine Glioma
• Pediatric Brainstem Glioma
• Pediatric Brainstem Gliosarcoma, Recurrent
• Pediatric Cancer
• Pediatric Brain Tumor
• Diffuse Glioma

Interventions

Type	Name	Description
DRUG	Marizomib	\- Intravenously initially given every other week over a 28 day course but will go to weekly x 3 and weekly x 4 as the dose levels increase Up to 26 courses.
DRUG	Panobinostat	\- Panobinostat: Oral dosage is given 3 times weekly, every other week over a 28 day course

Timeline

• Start date: 2020-08-10
• Primary completion: 2024-02-14
• Completion: 2024-02-14
• First posted: 2020-04-10
• Last updated: 2024-02-15

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study