A Planned Study Will Investigate the Effects of an Investigational Drug in Children with Glioma

A study is planned to investigate the effects of the experimental drug PTC596 in children with recently diagnosed diffuse intrinsic pontine glioma and high-grade glioma. The study is made up of two stages; phase I will investigate the recommended phase II dose, and phase II will involve a surgical component and assess the effects of PTC596. For more detailed information, you can read the source here, at ClinicalTrials.gov, which uses information provided by the Children’s Hospital Medical Center, Cincinnati.

Gliomas are brain tumours that can affect children and adults. You can learn more about glioma here.

The clinical trial is expected to recruit approximately thirty patients, who could be aged between 36 months to 21 years old.

Phase I

Phase I of the trial is designed to find the maximum tolerated dose or recommended dose of the experimental drug PTC596 for phase II. Patients taking part in this phase will also be treated with radiation therapy and maintenance therapy with the drug. According to the website, PTC596 will be given twice per week for six to seven weeks, and maintenance therapy will last for up to 25 cycles. For this first part of the trial, researchers will evaluate factors such as what the highest safe dose of the drug is (in combination with radiation therapy), the possible side effects of the drug, whether it affects patient quality of life, and whether the combination is an effective treatment, amongst other measures.

Phase II

After phase I finds out the recommended dose for phase II of PTC596, the second surgical phase of the study will enrol participants that have a newly-diagnosed DIPG and patients who have recently been diagnosed with HGG, and who also meet other eligibility criteria. Up to six patients are expected to take place on this surgical part of the study. Researchers will investigate how effective PTC596 is as a treatment (in combination with radiation therapy), and if it is able to reach tumours in the brain. In addition, the study aims to evaluate whether the drug affects patient quality of life and what the side effects of the drug may be, among other things.

To find out more about this study, how it is carried out, and who is eligible, you can look at the original study listing here (ClinicalTrials.gov Identifier: NCT03605550).


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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