Actively Recruiting
A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours
Led by Ipsen · Updated on 2026-05-06
6
Participants Needed
6
Research Sites
230 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate safety and the way the body absorbs, distributes and gets rid of the study drug tovorafenib in the body in Japanese children, adolescents and young adults with specific brain tumours. This includes how the drug is absorbed, distributed and eliminated from the body (called pharmacokinetics). The study will also test how well the drug works to shrink brain tumours. In this study, all participants will receive tovorafenib orally once weekly. There will be four periods in this study: 1. Screening period (up to 4 weeks): Participants will be evaluated to determine if they can take part in the study, requiring at least one visit to the study centre. 2. Treatment period (up to 24 months): All eligible participants will receive tovorafenib. This requires five visits for the first 2 months (Cycle 1 and Cycle 2) followed by one visit every month (at the start of each treatment cycle). Participants will receive the first oral dose of tovorafenib on Day 1 of Cycle 1 at the study clinic. After Day 1, participants will need to take tovorafenib once weekly on Day 8, Day 15 and Day 22. Participants will be required to come to the study clinic in person at least five times for Cycle 1 and Cycle 2. In addition, participants will have one remote visit (telephone call) during Cycle 1. After Cycle 2, only one in-person clinic visit is required at the start of each treatment cycle. A participant will stop treatment if their disease gets worse, if treatment has a harmful effect, or if they do not want to take part in the study anymore. 3. End-of-Treatment Safety Follow-Up (30 days): Participants will have a clinic visit 30 days after stopping treatment to check their health. 4. Long-Term Follow-Up (up to 2 years): Participants will be monitored every 3 months unless they start a new anti-cancer treatment or leave the study. During the study, participants will undergo various health measurements and observations, including blood sampling and urine collections. Each participant will be in this study for up to approximately 4 years. Tovorafenib will be provided to participants who tolerate it for as long as their disease does not progress. Once tovorafenib becomes approved and commercially available in Japan, participants may transition to the commercial drug for continued treatment. A participant may withdraw consent to participate at any time.
CONDITIONS
Official Title
A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 6 months to 25 years with at least two generations of Japanese ancestry
- Diagnosed with relapsed or progressive low-grade glioma with documented BRAF alteration
- Histopathologic confirmation of malignancy at diagnosis or relapse
- Received at least one prior systemic therapy with evidence of disease progression
- Have at least one measurable lesion by imaging within 28 days before treatment
- Fully recovered from acute toxic effects of prior anticancer therapy
- Stable chronic toxicities at NCI CTCAE Grade 2 or less; ongoing retinopathy Grade 1 or less
- Adequate blood, liver, and kidney function
- Stable steroid dose for tumor symptoms for 14 days before treatment
- Able to swallow tablets, liquid, or use feeding tube (12 Fr or greater)
You will not qualify if you...
- Tumor has additional activating molecular alterations besides BRAF
- Symptoms of clinical progression without imaging evidence of tumor progression
- Known or suspected neurofibromatosis type 1
- History of major diseases interfering with safe participation
- Retinal or eye conditions increasing risk of complications, excluding optic pathway glioma effects
- Major surgery within 14 days before treatment start, excluding certain procedures
- Significant active heart disease or recent severe cardiovascular events
- Moderate or worse nausea/vomiting, malabsorption, or major stomach/bowel surgery affecting drug absorption
- Neurologic instability despite treatment (e.g., uncontrolled seizures)
- Use of strong CYP2C8 inhibitors/inducers or narrow therapeutic index BCRP substrates within 14 days before therapy
- Significant skin toxicity at screening increasing risk with study drug
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
Kanagawa Children's Medical Center
Kanagawa, Japan
Not Yet Recruiting
2
Hyogo Prefectural Kobe Children's Hospital
Kobe, Japan
Actively Recruiting
3
Kyoto University Hospital
Kyoto, Japan
Actively Recruiting
4
Osaka City General Hospital
Osaka, Japan
Actively Recruiting
5
National Cancer Center Hospital
Tokyo, Japan
Actively Recruiting
6
National Center for Child Health and Development
Tokyo, Japan
Actively Recruiting
Research Team
I
Ipsen Clinical Study Enquiries
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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