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Phase I dose escalation and expansion trial of single agent ONC201 in pediatric diffuse midline gliomas following radiotherapy.

Sharon L Gardner,Rohinton S Tarapore,Jeffrey Allen,Susan L McGovern,Wafik Zaky,Yazmin Odia,Doured Daghistani,Zuanel Diaz,Matthew D Hall,Ziad Khatib,Carl Koschmann,Evan Cantor,Ryo Kurokawa,Tobey J MacDonald,Dolly Aguilera,Nicholas A Vitanza,Sabine Mueller,Cassie Kline,Guangrong Lu,Joshua E Allen,Soumen Khatua

Neuro-Oncology Advances（2022）

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摘要
The adult 625 mg weekly RP2D of ONC201 scaled by body weight was well tolerated. Further investigation of ONC201 for DMG/DIPG is warranted.
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关键词
H3 K27M-mutant glioma (DMG),ONC201,diffuse intrinsic pontine glioma (DIPG),diffuse midline glioma
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