Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma

This research study is investigating a new drug called Vorasidenib alongside standard chemotherapy, Temozolomide (TMZ), for people with a specific type of brain tumour called IDH-mutant glioma. The study has two main parts: first, it will carefully check the safety of the new drug combination and find the best dose to use. Once the safest dose is known, the second part of the study will see how effective this combination is at treating the brain tumour. Participants will have regular clinic visits, blood tests, and scans, and will be monitored for a long time after treatment ends to see how they are doing. The goal is to find better ways to treat this condition.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Institut de Recherches Internationales Servier

Enrolment target

Start

22 Jan 2025

Estimated completion

01 Jun 2028

IDH1-mutant Glioma IDH2-mutant Glioma

What is this study about?

This study is looking at a new drug called Vorasidenib for people with a specific type of brain tumour known as IDH-mutant glioma. This type of glioma has a particular change in its genes (either IDH1 or IDH2). Vorasidenib will be given together with Temozolomide (TMZ), which is a common chemotherapy drug used to treat brain tumours.

The main aim of this research is to see if combining Vorasidenib with TMZ is safe and well-tolerated by patients, and to work out the best dose of Vorasidenib to use. Once doctors are happy with the safety and dose, the study will then look at how effective this new combination is at treating the tumour.

Finding new and better treatments for brain tumours is very important. Studies like this help us understand if new drugs can improve outcomes for patients when used alone or with existing treatments. This study could help doctors learn more about how to manage IDH-mutant glioma in the future.

Key takeaways

This study combines a new drug (Vorasidenib) with a standard chemotherapy (TMZ).
It's for people with a specific type of brain tumour called IDH-mutant glioma.
The study first checks for safety and the best dose, then how effective it is.
Participants will have regular visits, blood tests, and long-term follow-up.
The aim is to find better treatments for this brain tumour.
You can stop participating at any time.

Who may be eligible?

To join this study, you need to be at least 12 years old and weigh at least 40 kg. You must have a confirmed IDH1 or IDH2 gene change in your brain tumour, and your general health, including your kidney, liver, and blood counts, needs to be good enough for you to take part safely. Doctors also need to think you have a life expectancy of at least three months.

If you are female and could become pregnant, you'll need a negative pregnancy test before starting treatment. If you are taking steroid medication for your brain tumour, your dose needs to be stable or decreasing and at a low level for at least five days before starting the study.

For the first part of the study, you'll also need a specific type of astrocytoma or oligodendroglioma, and your doctor must believe that treatment with Temozolomide is appropriate for you. If you've recently had radiotherapy, you would need to start study treatment within six weeks of finishing it.

Quick self-check

Are you 12 years of age or older and weigh at least 40 kg?
Has your brain tumour been confirmed to have an IDH1 or IDH2 gene change?
Are your kidney, liver, and blood test results generally healthy enough?
If you are female, are you able to have a negative pregnancy test?
Are you able to start Temozolomide treatment, according to your doctor?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you will be given Vorasidenib alongside Temozolomide. During the treatment period, you will have study visits on the first and 22nd day of each treatment cycle. In the very first cycle, you might have a few extra visits. These visits will involve various checks, such as filling out questionnaires, having blood tests, getting an ECG (a heart tracing), checking your vital signs (like blood pressure and pulse), and having a physical examination by a doctor.

After you finish your treatment, you'll have a safety follow-up visit about a month later. Following that, doctors will continue to check in with you every three months to see how you are doing, usually for quite a long time, to monitor your health and survival. The total duration of your active treatment will depend on how you respond, but the follow-up period will be ongoing.

Potential risks and benefits

Participating in this study might offer the potential benefit of accessing a new treatment (Vorasidenib) in combination with a standard one, which could be helpful for your condition. However, there are potential risks, as both drugs can have side effects, which your medical team will explain in detail. Because this is a research study, the doctors are still learning about this new combination, so not all risks might be fully known. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (28)

University of California Los Angeles
Los Angeles, United States
University of Miami
Miami, United States
Dana Farber Cancer Institute
Boston, United States
Memorial Sloan Kettering Cancer Center
New York, United States
Duke University Medical Center
Durham, United States
MD Anderson Cancer Center
Houston, United States
Medical University of Vienna - AKH
Vienna, Austria
Beijing Tiantan Hospital, Capital Medical University
Beijing, China
Huashan Hospital, Fudan University
Shanghai, China
Hôpital Pierre Wertheimer
Lyon, France
Hôpital Pitié-Salpêtrière
Paris, France
IUCT-Oncopole Institut Universitaire du Cancer
Toulouse, France

+16 more sites — see the official record for the full list.

Common questions

What is an 'IDH-mutant glioma'?

It's a type of brain tumour that has a specific genetic change (mutation) in either the IDH1 or IDH2 gene.

What is Vorasidenib?

Vorasidenib is a new drug being tested in this study. It's designed to target specific cancer cells.

What is Temozolomide (TMZ)?

TMZ is a standard chemotherapy drug commonly used to treat brain tumours.

What is a 'Phase 1b' and 'Phase 2' study?

Phase 1b checks for safety and the best dose, and Phase 2 then looks at how well the treatment works.

Will I receive both drugs?

Yes, if you join this study, you will receive Vorasidenib in combination with Temozolomide.