In short
Clinical trials are studying Vorasidenib in people with certain IDH-mutant brain tumors. These studies look at how well it works, how safe it is, and whether it helps delay tumor growth in different patient groups, including those after surgery or after chemoradiotherapy.
Key points
- Clinical trials of Vorasidenib are studying people with IDH-mutant brain tumors, mainly glioma and astrocytoma. Two Phase 3 studies compare Vorasidenib with placebo in patients with residual or recurrent grade 2 glioma after surgery only, and in patients with grade 2 or 3 astrocytoma after chemoradiotherapy. A Phase 1 study is testing Vorasidenib with temozolomide in people with IDH1- or IDH2-mutant glioma. The main outcomes include progression-free survival, radiographic progression-free survival, and safety measures such as side effects and serious side effects. These trials are designed to see whether Vorasidenib can help delay tumor growth in specific patient groups.
Trial overview
These studies are looking at Vorasidenib in people with specific IDH1 or IDH2 mutated brain tumors. The trials focus on glioma and astrocytoma, including lower-grade and higher-grade disease, and they are designed to test whether the treatment can delay tumor growth and how safe it is.
All of the trials listed are interventional studies, which means the researchers give a treatment and then measure the results. The studies include two Phase 3 trials and one Phase 1 trial.
Who can join the studies
Each trial has a different target group, but all are based on tumor type and mutation status. In one study, people must have residual or recurrent grade 2 glioma with an IDH1 or IDH2 mutation and must have had surgery as their only treatment.
Another Phase 3 study includes people with IDH-mutant grade 2 or 3 astrocytoma after first-line chemoradiotherapy, which means after a first round of chemotherapy and radiation treatment. The Phase 1 study includes participants with IDH1- or IDH2-mutant glioma and tests Vorasidenib together with temozolomide.
Trial phases and study design
The two Phase 3 studies are larger trials meant to test whether Vorasidenib works better than placebo in the chosen patient groups. A placebo is a look-alike treatment without active medicine, used so the results can be compared fairly.
The Phase 1 study is smaller and focuses first on safety and tolerability, which means how well people can take the treatment and what side effects happen. It also explores early signs that the combination with temozolomide may help control the disease.
What the trials measure
The main outcome in the first Phase 3 study is radiographic progression-free survival, measured by a blinded review committee using modified RANO-LGG criteria. This means the researchers use scans to see how long the tumor does not get worse.
The second Phase 3 study measures progression-free survival from the date of enrollment using RANO 2.0 criteria. The goal is to show that Vorasidenib maintenance therapy can improve the time before the disease gets worse compared with placebo.
The Phase 1 study measures dose-limiting toxicities in the Phase 1b part, along with the incidence and severity of adverse events, serious adverse events, and adverse events of special interest. It also looks at progression-free survival status at 12 months and early clinical efficacy in the group treated at the recommended dose.
Key studies of Vorasidenib
NCT04164901 is a Phase 3 study in residual or recurrent grade 2 glioma with an IDH1 or IDH2 mutation. Its main goal is to show that Vorasidenib improves radiographic progression-free survival compared with placebo in people who had surgery as their only prior treatment.
NCT06809322 is a Phase 3 study in IDH-mutant grade 2 or 3 astrocytoma after standard chemoradiotherapy. It studies Vorasidenib as maintenance therapy and compares it with placebo to see whether it improves progression-free survival.
NCT06478212 is a Phase 1 multicenter study of Vorasidenib with temozolomide in participants with IDH1- or IDH2-mutant glioma. This study is mainly about safety, tolerability, and early signs of benefit, and it also includes a 12-month progression-free survival check.
Patient-focused terms
Progression-free survival means the time during which the tumor does not grow or get worse. Adverse events are unwanted medical problems that happen during a study, and serious adverse events are the more severe ones.
Recommended dose means the dose chosen for later study after safety testing. Multicenter means the study is run at more than one hospital or clinic.