Study of tovorafenib (DAY101) in children and young adults aged 6 months to 25 years with brain tumors and other solid tumors that have RAF gene changes

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What is this study about?

This study focuses on children and young adults with low-grade glioma (a type of brain tumor) and other advanced solid tumors that have specific genetic changes called RAF alterations. The study will test a new medication called tovorafenib (also known as DAY101), which is designed to target these specific genetic changes in tumors. This medicine comes in two forms – as a powder that can be made into a liquid for drinking, and as tablets.

The purpose of this research is to determine how well tovorafenib works in treating these types of tumors and to understand its safety in patients aged 6 months to 25 years. The medication is specifically designed to reach the central nervous system (brain and spinal cord) and works by blocking certain proteins that can cause tumor growth.

During the study, participants will receive tovorafenib daily by mouth. The treatment may continue for up to 72 months, depending on how well it works for each patient. Doctors will regularly check the size of tumors using imaging tests and monitor patients for any side effects that may occur during treatment.

1 Initial evaluation

Your eligibility for the study will be assessed based on your age (between 6 months and 25 years) and medical condition.

A confirmation of your brain tumor (low-grade glioma) or advanced solid tumor diagnosis is required.

Your previous treatments and disease progression will be reviewed.

Your physical condition will be evaluated using a performance score test.

2 Treatment assignment

You will be assigned to one of three treatment groups:

Group 1: For patients with low-grade glioma with specific genetic changes

Group 2: For patients with low-grade glioma with certain genetic alterations

Group 3: For patients with advanced solid tumors with specific genetic changes

3 Medication administration

You will receive tovorafenib (also known as DAY101) in one of two forms:

Either as a powder that will be mixed into a liquid for drinking

Or as tablets that you will take by mouth

Your doctor will provide specific instructions about how and when to take the medication

4 Monitoring period

Regular check-ups will track how your body responds to the treatment

Your tumor size will be measured regularly using imaging scans

Blood tests will be performed to monitor how your body processes the medication

Any side effects or health changes will be recorded

5 Study completion

The study will continue until April 2027

Your participation may end earlier based on how your condition responds to the treatment

A final evaluation will be performed to assess the overall results of your treatment

Who Can Join the Study?

Patient must be between 6 months and 25 years old
Patient must have one of these conditions:
- Returning or worsening low-grade glioma (a slow-growing brain tumor) with a confirmed BRAF alteration (a specific genetic change)
- Returning or worsening low-grade glioma with a confirmed or expected BRAF mutation or RAF fusion (specific types of genetic changes)
- Advanced or spreading solid tumor with a confirmed or expected RAF fusion
Patient must have had their cancer confirmed through tissue examination (biopsy) either when first diagnosed or when the cancer returned
Patient must have received at least one previous treatment and show evidence that the cancer has grown on imaging scans (for low-grade glioma patients only)
Patient must have at least one tumor that can be measured or evaluated by medical imaging
Patient must have a performance score of at least 50 on either:
- The Karnofsky scale (for patients 16 years and older) – a scale that measures a person’s ability to perform daily activities
- The Lansky scale (for patients under 16 years) – a similar scale used for children

Who Cannot Join the Study?

Age below 6 months or above 25 years
Absence of RAF gene alterations (changes in specific genes that affect cell growth)
No evidence of tumor progression or recurrence
Previous treatment with tovorafenib (the study medication)
Patients who have not experienced a relapse or progression of their low-grade glioma (slow-growing brain tumor) or advanced solid tumor
Patients who cannot undergo required medical imaging to monitor their condition
Inability to take oral medications
Presence of other serious medical conditions that could interfere with study participation
Current participation in other clinical trials
Pregnancy or breastfeeding
Known allergies or hypersensitivity to the study medication or its components

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Rigshospitalet	Copenhagen	Denmark

Trial status

Country	Status	Recruitment Start
Denmark	Recruiting	01.04.2021
Germany	Recruiting	01.04.2021
The Netherlands	Recruiting	01.04.2021

Trial locations

Investigated drugs:

Tovorafenib

Tovorafenib (also known as DAY101) is an oral medication that works as a pan-RAF inhibitor. It targets and blocks specific proteins called RAF that are involved in cancer cell growth. This medication is designed to treat pediatric patients who have certain types of brain tumors called low-grade gliomas and other advanced solid tumors where the RAF genes have been altered. The medication works by interfering with abnormal cell signaling that causes tumor growth, particularly in cases where there are BRAF alterations or RAF fusions.

Investigated diseases:

Malignant glioma

Low-Grade Glioma – A type of brain tumor that develops from glial cells and grows slowly in the brain or spinal cord. It begins in supportive brain tissue and typically has a less aggressive growth pattern compared to high-grade variants. These tumors can affect various parts of the brain, leading to different symptoms depending on their location. Low-grade gliomas can gradually increase in size over time, potentially causing pressure on surrounding brain structures.

Advanced Solid Tumor – A solid mass of abnormal cells that can occur in various body tissues and organs, which has spread beyond its original location. These tumors form distinct masses or lumps that can be detected in tissue. Solid tumors can develop in different parts of the body, including organs, muscles, or bones. They are characterized by the growth of abnormal cells that multiply uncontrollably and can expand into nearby tissues.

Trial ID:

2024-510691-20-00

Protocol code:

DAY101-001

NCT ID:

NCT04775485

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of tovorafenib (DAY101) in children and young adults aged 6 months to 25 years with brain tumors and other solid tumors that have RAF gene changes

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?