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Clinical trials for diffuse intrinsic pontine glioma: the current state of affairs

  • Julian S. Rechberger
  • Victor M. LuEmail author
  • Liang Zhang
  • Erica A. Power
  • David J. DanielsEmail author
Review Article

Abstract

Purpose

Diffuse intrinsic pontine glioma (DIPG) is a lethal high-grade pediatric brainstem tumor without a cure. Despite numerous clinical trials over the last decades, the prognosis has remained poor. The aim of this update was to report on the status and outcomes of all clinical trials for DIPG performed to better understand the landscape of research efforts for this diagnosis to date.

Methods

The ClinicalTrials.gov database was reviewed in May 2019 for all possible interventional clinical trials that included DIPG as a diagnosis of primary investigation. These were then screened against selection criteria to identify pertinent clinical trials.

Results

Ninety-five clinical trials satisfied all inclusion criteria, with 55 (58%) trials specific to the DIPG diagnosis only. In terms of the most prevalent design features, 42 (44%) were phase I trials, with median expected start and completion years in 2011 (range, 1994–2020) and 2018 (range, 2005–2047), respectively. Median target number of patients to enroll was 38 (range, 1–1500), and the most common primary outcome was safety and toxicity (56%). There were 69 (73%) trials originating from the USA, with 49 (52%) of them being single institutional. Only 10 (11%) trials have reported results to date.

Conclusions

To date, 95 clinical trials investigating DIPG with specific emphasis have been registered on ClinicalTrials.gov. There were only a small number of trials that had study results available, and they uniformly reported non-significant improvement to prognosis. Given the rarity and lethality of DIPG, which limits the accumulation of large cohorts, our results mandate the need for more robust, systematic clinical trial design to minimize redundancies and maximize yield in the future.

Keywords

Diffuse intrinsic pontine glioma DIPG H3 K27M Clinical trial Phase I Randomized 

Notes

Compliance with ethical standards

Conflict of interest

The authors report no funding sources or conflict of interest concerning the materials or methods used in this study or the findings specified in this study.

Supplementary material

381_2019_4363_MOESM1_ESM.docx (73 kb)
ESM 1 (DOCX 72 kb)

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Copyright information

© Springer-Verlag GmbH Germany, part of Springer Nature 2019

Authors and Affiliations

  1. 1.Department of Neurologic SurgeryMayo ClinicRochesterUSA
  2. 2.Mayo Clinic Graduate School of Biomedical SciencesMayo ClinicRochesterUSA

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