NCT07441707

A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours

Not yet recruiting Phase 1 Last updated 2 March 2026

What this trial tests

Phase 1 trial testing Tovorafenib in Low-grade Glioma in 6 participants. Not yet recruiting.

Timeline

16 February 2026

Primary endpoint
31 July 2030

31 July 2030

Quick facts

Lead sponsor	Ipsen
Phase	Phase 1
Status	Not yet recruiting
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	6
Start date	16 February 2026
Primary completion	31 July 2030
Estimated completion	31 July 2030

Drugs / interventions tested

• Tovorafenib (TOVORAFENIB) — full drug profile →

Conditions studied

• Low-grade Glioma — all drugs for Low-grade Glioma →

Sponsor

Ipsen — full company profile →

Who can join

Adults 6 Months to 25, any sex, with Low-grade Glioma. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.