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Gadolinium-chelated polysiloxane nanoparticles are an investigational substance containing a metal called gadolinium attached to nanoparticles.[2] The substance is not an authorised medicine; it was designated as an orphan medicine for the treatment of glioma in the European Union on 14 January 2022, which means the developer receives scientific and regulatory support from the European Medicines Agency (EMA) but the medicine is not yet available for use outside clinical trials.[2] In the trial registry, the substance appears under the name AGuIX.[1]
The medicine contains gadolinium, a metal, attached to nanoparticles. These metallic nanoparticles are expected to enter cancer cells and help the tumour absorb more radiation during radiotherapy.[2] They are also expected to cause the formation of very reactive chemicals called reactive oxygen species, which can further damage the cancer cells.[2] This combined effect is expected to slow down the progression of the disease.[2]
The substance is given by intravenous administration, meaning it is injected directly into a vein.[1] In the clinical trial, it is given in combination with radiotherapy and temozolomide, a chemotherapy medicine, during the concomitant radiochemotherapy period.[1]
One clinical trial is currently authorised for this substance. It is a Phase 1/2 study with a planned enrolment of 66 participants with newly diagnosed glioblastoma.[1] The trial investigates AGuIX nanoparticles given together with radiotherapy and concomitant temozolomide.[1]
The study is divided into two parts. Phase I aims to determine the recommended dose of AGuIX in combination with radiotherapy and temozolomide during the concomitant radiochemotherapy period.[1] The recommended dose is defined as the highest dose tested in which the percentage of dose-limiting toxicities is less than 33%; a dose-limiting toxicity is defined as any grade 3–4 toxicity, except alopecia, nausea, and vomiting, occurring during the first 6 weeks.[1] Phase II aims to estimate the efficacy of the combination radiochemotherapy plus AGuIX at the recommended dose, measured by the 6-month progression-free survival rate using RANO criteria.[1] Progression-free survival means the length of time a participant lives without the cancer getting worse.
At the time of the orphan designation application, the effects of the medicine had been evaluated in experimental models, and clinical trials with the medicine in patients with glioma were ongoing.[2]
The supplementary source does not report specific side effects of gadolinium-chelated polysiloxane nanoparticles. The clinical trial will collect information on dose-limiting toxicities, defined as any grade 3–4 toxicity except alopecia, nausea, and vomiting, during the first 6 weeks of treatment.[1]
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