At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Tovorafenib (drug)
- How long the study runs
- Study runs about 73 months (dates as stated)
- About the drug or intervention
- Tovorafenib — drug: Tovorafenib is an oral Type II RAF kinase inhibitor available in 100 mg immediate-release tablet or 25 mg/milliliter (mL) powder for reconstitution.
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is looking at a medicine called tovorafenib in children, teenagers and young adults whose low-grade glioma (a slow-growing brain tumour) has come back or got worse, or who have an advanced solid tumour. It is paid for (sponsored) by Day One Biopharmaceuticals, Inc.
Who can take part
- For low-grade glioma: the tumour has come back or grown and has a known change (alteration) in a gene called BRAF that switches it on.
- For advanced solid tumours: the tumour has spread or grown locally and has a known or expected RAF gene fusion (when two genes join together in an abnormal way).
- The cancer has been confirmed by examining tissue under a microscope, either when first diagnosed or when it came back.
- The person has already had at least one course of medicine that treats the whole body, and scans show the tumour has grown.
- The person has at least one tumour that can be measured on a scan, using standard measuring rules called RANO (arms 1 and 2) or RECIST version 1.1 (arm 3).
Who may not be able to
- The tumour has other known gene changes that switch cancer on, found before joining the study.
- The person has symptoms but scans do not show the tumour has come back or grown.
- The person has, or may have, a condition called neurofibromatosis type 1 (NF-1), found by genetic testing or current diagnosis rules.
- Other rules in the study plan may also apply — the trial team can explain these.
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask your trial team about how many visits are needed, how long the study lasts, and what taking part would involve.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 6 Months to 25 Years
- Who
- All
- Number of participants
- 141
- Started
- 2021-04-22
- Last checked
- 2025-03
Plain English Summary
What is this study?
- • Testing a new treatment for low-grade glioma
- • Phase2 - 141 participants
- • This is a Phase 2, multi center, open-label study to evaluate the safety and efficacy of Type II RAF (tovorafenib) in pediatric participants with low-grade glioma or advanced solid tumors
Who can take part?
- • Ages 6 Months to 25 Years
- • Diagnosed with low-grade glioma
Where?
- • London - UCL Great Ormond Street Institute of Child Health
- • Newcastle upon Tyne - Newcastle University
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a Phase 2, multi center, open-label study to evaluate the safety and efficacy of Type II RAF (tovorafenib) in pediatric participants with low-grade glioma or advanced solid tumors. Qualifying genomic alterations will be identified through molecular assays as routinely performed at Clinical Laboratory Improvement Amendments (CLIA) of 1988 or other similarly certified laboratories prior to enrollment into any of the arms. The study will consist of a screening period, a treatment period, a long-term extension phase, end of treatment (EOT) visit(s), a safety follow-up visit, and long-term follow-up assessments.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 6 Months - 25 Years
- Who can join: All genders
Biomarkers mentioned
Treatment history
Treatments you must have had:
- ✓ histopathologic verification of malignancy at either original diagnosis or relapse
- ✓ at least 1 measurable lesion as defined by RANO (Arms 1 \& 2) or RECIST v1
What the study is looking for
- ✓Low Grade Glioma \& Low-Grade Glioma Extension: a relapsed or progressive LGG with documented known activating BRAF...
- ✓Advanced Solid Tumor: that has grown locally or that has spread solid tumor with documented known or expected to be activating...
- ✓Participants must have histopathologic verification of cancer at either original diagnosis or relapse.
- ✓Must have received at least one line of prior treatment that goes through your whole body and have documented evidence of radiographic progression.
- ✓Must have at least 1 measurable lesion as defined by RANO (Arms 1 \& 2) or (standard scan measurements) (Arm 3) criteria
Who cannot take part
- ✗Participant's tumor has additional previously-known activating molecular alterations.
- ✗Participant has symptoms of without radiographically recurrent or radiographically progressive disease.
- ✗Known or suspected diagnosis of neurofibromatosis type 1 (NF-1) via genetic testing or current diagnostic criteria.
- ✗Other inclusion/exclusion criteria as stipulated by protocol may apply
See the full criteria
Where Is This Study? (2 UK sites)
UCL Great Ormond Street Institute of Child Health
London WC1N 1EH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700Newcastle University
Newcastle upon Tyne NE1 7RU, United Kingdom
How to Get in Touch
Day One Biopharmaceuticals, Inc.
Sponsor contactCONTACT