RecruitingPHASE2INTERVENTIONAL

Study of Olutasidenib and Temozolomide in HGG

This research study is looking at a new treatment for young people (aged 12-39) with a specific type of brain tumour called high-grade glioma (HGG). This cancer must have a particular genetic change, known as an IDH1 mutation. The study aims to see if combining a new medicine, olutasidenib, with temozolomide (a standard chemotherapy drug) can help treat this brain tumour more effectively after patients have had radiotherapy. The main goal is to find out if this combination treatment can help patients live longer. Researchers will also look at how effective the treatment is, what side effects it might cause, and how the body handles the new drug. It's a phase 2 study, meaning it's comparing the new treatment to how similar patients have done in the past, to see if it's promising enough for larger studies.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Rigel Pharmaceuticals

Enrolment target

Start

01 Mar 2025

Estimated completion

01 Jun 2035

High Grade Glioma Astrocytoma Astrocytoma, Grade III Astrocytoma, Grade IV Diffuse Intrinsic Pontine Glioma WHO Grade III Glioma WHO Grade IV Glioma Metastatic Brain Tumor Diffuse Midline Glioma, H3 K27M-Mutant Thalamus Tumor Spinal Tumor IDH1 Mutation IDH1 R132 IDH1 R132C IDH1 R132H IDH1 R132S IDH1 R132G IDH1 R132L Oligodendroglioma

What is this study about?

This study is focused on a type of brain tumour known as high-grade glioma (HGG), which includes several kinds of aggressive brain cancers. It's specifically for young people, aged 12 to 39, whose tumours have a particular genetic change called an IDH1 mutation. This mutation is like a specific 'marker' in the cancer cells that the new drug, olutasidenib, is designed to target.

The main purpose of this research is to see if combining olutasidenib with a standard chemotherapy drug called temozolomide (TMZ) can improve the treatment for these patients. Patients in the study will have already completed their initial radiotherapy, which is a common treatment for these tumours. After radiotherapy, they will receive the combination of olutasidenib and TMZ. The researchers want to know if this new approach can help patients live longer without their tumour growing back.

This is a 'Phase 2' study, which means it's one of the earlier stages of drug development. The researchers are also looking at how safe the new drug combination is, what side effects might occur, and how well the body absorbs and uses olutasidenib. They will compare the results of patients in this study to historical information from similar patients treated in the past, to understand the potential benefits of this new treatment strategy.

Key takeaways

This study is for young people (12-39) with a specific type of brain tumour called high-grade glioma (HGG).
Your tumour must have a particular genetic change called an IDH1 mutation to be eligible.
It tests a new drug, olutasidenib, combined with standard chemotherapy (temozolomide) after radiotherapy.
The main aim is to see if this combination can help patients live longer without their tumour growing.
Participation involves regular hospital visits for treatment, monitoring, and health checks.
You can stop participating in the study at any time for any reason.

Who may be eligible?

To join this study, you must be between 12 and 39 years old and have been newly diagnosed with a high-grade glioma (HGG) brain tumour. Your tumour must have a specific genetic change called an IDH1 mutation. This includes certain types of brain tumours like astrocytomas, as well as a specific type called Diffuse Intrinsic Pontine Glioma (DIPG) that affects the brainstem, or tumours in the thalamus or spinal cord.

Your doctors will need to confirm the exact type of your tumour and the presence of the IDH1 mutation through a biopsy or surgery tissue sample. You cannot have cancer that has spread to other parts of your body, and your tumour should not have already been treated in certain ways. For example, if you have a specific type of brain tumour called oligodendroglioma with an IDH mutation and a 1p/19q co-deletion (another genetic change), you would not be able to participate. Also, if your tumour has an IDH2 mutation instead of an IDH1 mutation, you would not be eligible.

Quick self-check

Are you between 12 and 39 years old?
Have you been newly diagnosed with a high-grade glioma (brain tumour)?
Has your doctor confirmed that your tumour has an IDH1 genetic mutation?
Do you have localised cancer (not spread to other parts of the body)?
Have you already completed initial radiotherapy for your brain tumour?

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

What does participation involve?

If you join the study, you'll receive the study drug olutasidenib along with temozolomide (TMZ) after you've finished your initial radiotherapy. This combination treatment will be given for 13 cycles (rounds). After these 13 cycles, you'll continue to receive just olutasidenib for another 13 cycles. During this time, you'll have regular hospital visits to monitor your health, check for side effects, and see how the treatment is working. This will likely involve blood tests, scans, and doctor assessments. The total treatment period for the study medications is about two years, and you will have follow-up appointments after this to continue monitoring your health and the long-term effects of the treatment.

Potential risks and benefits

Participating in this study might offer the potential benefit of trying a new treatment that could be more effective for your specific type of brain tumour than current standard options. However, like all medications, olutasidenib and temozolomide can have side effects, which the study team will monitor closely. There's also no guarantee that the treatment will be effective for everyone. You will be fully informed about all known risks before deciding to join, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (18)

Children's Hospital Colorado
Aurora, United States· Not yet recruiting
Children's National Medical Center
Washington D.C., United States· Not yet recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States· Not yet recruiting
Susan Chi
Boston, United States· Not yet recruiting
Duke University Health System
Durham, United States· Not yet recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, United States· Recruiting
Nationwide Children's Hospital
Columbus, United States· Not yet recruiting
Children's Hospital of Philadelphia
Philadelphia, United States· Not yet recruiting
Texas Children's Hospital
Houston, United States· Not yet recruiting
Seattle Children's Hospital
Seattle, United States· Not yet recruiting
Sydney Children's Hospital
Randwick, Australia· Not yet recruiting
Queensland Children's Hospital
South Brisbane, Australia· Not yet recruiting

+6 more sites — see the official record for the full list.

Common questions

What is an 'IDH1 mutation'?

It's a specific change in the DNA of your tumour cells. This specific change is important because the new drug, olutasidenib, is designed to target tumours that have it.

What does 'high-grade glioma' mean?

High-grade glioma is a general term for certain types of aggressive brain tumours that tend to grow more quickly. The study includes several of these types.

What is a 'Phase 2 study'?

Phase 2 means it's an early stage of testing a new drug. The main goals are to see how safe the drug is, what dose works best, and if it shows encouraging signs of effectiveness for the disease.

Will I still receive my normal treatments if I join?

Yes, you would still have received radiotherapy, which is a standard treatment. This study combines the new drug with an existing chemotherapy, temozolomide, as a follow-up.

What if I experience side effects during the study?

The study team will closely monitor you for any side effects. They will provide care and guidance to manage them and ensure your safety throughout your participation.

How to find out more

Jill DeFratis Robinson

jrobinson@rigel.com 650-624-1100 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.