Clinical Trial: Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication

Brief Summary:

Diffuse Intrinsic Pontine Gliomas (DIPG) appear almost exclusively in children and adolescents, representing 15 to 20% of posterior fossa tumours. Even if it is one of the most common malignant brain tumours, there are only 30 to 40 new cases per year in France. Their clinical presentation is stereotyped with a short clinical history and a unique MRI appearance that was usually considered as sufficient to establish the diagnosis. The prognosis of DIPG is always unfavourable; median overall survival is 9 to 10 months in general and most patients will die within two years after diagnosis (Kaplan 1996,Hargrave 2006). Malignant gliomas infiltrating the brainstem represent the greatest challenge of paediatric oncology; despite numerous collaborative studies performed, patients' survival has not significantly increased in thirty years (Hargrave 2009). There is no validated prognostic factor. There is currently no validated treatment except radiotherapy.

Several targeted agents have been tested in DIPG (Pollack 2007 Haas-Kogan 2008, Geoerger, 2011), without knowing whether the target was present in the tumour. A critical review of the paradigms of these trials tells us that there are long term survivors in these studies that is to say patients who may have benefited from the tested therapy, but they are few. So far, the new therapies that have been tried were evaluated one after the other in search of a treatment that would be effective for all patients, measuring the treatment effect on median survival. They were all rejected as ineffective. However the investigators can challenge the endpoint to evaluate efficacy in these trials as the existence of long term survivors (> 18 months, for example) and their number should not been ignored, especially if targeted therapies are considered. The investigators propose a paradigm shift in the choice of treatment; the issue raised wou

Detailed Summary:
Sponsor: Gustave Roussy, Cancer Campus, Grand Paris

Current Primary Outcome: Overall Survival [ Time Frame: Assessed up two years after randomization ]

Original Primary Outcome: 2 years Overall Survival [ Time Frame: Assessed up two years after randomization ]

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Gustave Roussy, Cancer Campus, Grand Paris

Dates:
Date Received: August 29, 2014
Date Started: October 2014
Date Completion: October 2018
Last Updated: June 8, 2016
Last Verified: June 2016