Study of tovorafenib versus standard chemotherapy as first treatment for children with low-grade glioma with RAF gene changes

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What is this study about?

This study focuses on pediatric low-grade glioma, a type of brain tumor that occurs in children and young people under 25 years of age. The study specifically looks at cases where the tumor has certain genetic changes called RAF alterations and requires treatment for the first time. The purpose is to compare two different treatment approaches: a new medication called tovorafenib versus standard chemotherapy treatments that include vincristine, vinblastine, and carboplatin.

The study will determine which treatment is more effective at shrinking or controlling the tumor. Patients will be randomly assigned to receive either tovorafenib tablets or oral suspension taken by mouth, or standard chemotherapy medications given through an intravenous line (a small tube inserted into a vein). The study will measure how well tumors respond to treatment and how long patients remain free from tumor growth.

Doctors will regularly monitor patients using brain scans and medical examinations to track the tumor’s response to treatment. The study will continue for several years to gather long-term information about how well each treatment works and how it affects patients’ health over time. This research aims to help determine the most effective treatment option for children and young people with this specific type of brain tumor.

1 Initial evaluation and assignment

Your eligibility for the trial will be confirmed based on being under 25 years of age and having a low-grade glioma (a type of brain tumor) with specific genetic characteristics.

You will need to have at least one tumor that can be measured.

You will be randomly assigned to receive either the new medication or standard treatment.

2a Treatment path A – New medication

If assigned to this group, you will receive tovorafenib.

This medication will be taken by mouth, either as tablets or as a powder mixed with liquid.

2b Treatment path B – Standard treatment

If assigned to this group, you will receive standard chemotherapy medications:

Vincristine, vinblastine, and carboplatin will be given through an IV line.

These medications will be administered at regular intervals according to the standard treatment schedule.

3 Monitoring during treatment

Regular imaging scans will be performed to measure how your tumor responds to treatment.

Your response to treatment will be evaluated using specific criteria that look for any changes in tumor size.

Your overall health status will be monitored throughout the study.

4 Long-term follow-up

The study will continue until March 2030.

You will be monitored for any changes in your condition.

The medical team will track how long the treatment remains effective and your overall health status.

Who Can Join the Study?

  • Must be under 25 years old and have a low-grade glioma (a slow-growing brain tumor) with specific genetic changes in the RAF gene
  • Must have a confirmed diagnosis of glioma or glioneuronal tumor (types of brain tumors) through tissue examination
  • Must have at least one tumor that can be measured by imaging tests
  • Must be starting treatment for the first time with medication that goes throughout the body (systemic therapy)
  • Both males and females can participate in the study
  • Children and young people from birth to 24 years old are eligible

Who Cannot Join the Study?

  • Patients who do not have a confirmed diagnosis of pediatric low-grade glioma (a type of slow-growing brain tumor)
  • Patients whose tumor does not have an activating RAF alteration (a specific genetic change in the tumor)
  • Patients who have already received systemic therapy (medicine that travels through the bloodstream)
  • Patients younger than 6 months or older than 25 years of age
  • Patients who have received previous treatment with tovorafenib or similar medications
  • Patients with severe heart, liver, or kidney problems
  • Patients who are currently participating in other clinical trials
  • Patients who are pregnant or breastfeeding
  • Patients who cannot undergo required medical tests or procedures
  • Patients with known allergies to the study medications
  • Patients who have had major surgery within 4 weeks before starting the study
  • Patients who cannot swallow oral medications

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Universitaet Innsbruck Innsbruck Austria
Institut Gustave Roussy Villejuif France
Oslo Universitetssykehus HF Oslo Norway
Medizinische Hochschule Hannover Hanover Germany
Hospital Universitario Y Politecnico La Fe Valencia Spain
Medical University Of Vienna Vienna Austria
Universitaetsmedizin Goettingen Goettingen Germany
Universitaetsklinikum Heidelberg AöR Heidelberg Germany
Universitaet Leipzig Leipzig Germany
Katholieke Universiteit te Leuven Leuven Belgium
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari Bari Italy
Institut Curie – Site Paris Paris France

Other Sites

Site Name City Country Status
Universitetssykehuset Nord-Norge HF Tromsø Norway
IRCCS Istituto Giannina Gaslini Genoa Italy
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Universitair Ziekenhuis Gent Gent Belgium
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Azienda Sanitaria Universitaria Friuli Centrale Udine Italy
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Nosokomeio Paidon I Agia Sofia Athens Greece
Azienda Ospedaliera Santobono Pausilipon Naples Italy
Azienda Ospedaliera di Padova Padua Italy
Athens General Children’s Hospital Panagioti And Aglaia Kyriakou Athens Greece
Virgen del Rocío University Hospital Sevilla Spain
Universitaetsklinikum Erlangen AöR Erlangen Germany
Universitaetsklinikum Tuebingen AöR Tuebingen Germany
Region Skane Skanes Universitetssjukhus Lund Sweden
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
Karolinska University Hospital Solna Sweden
Evangelisches Klinikum Bethel gGmbH Bielefeld Germany
Semmelweis University Budapest Hungary
Hospital Universitario De Cruces Barakaldo Spain
Prinses Maxima Centrum voor Kinderoncologie B.V. Utrecht The Netherlands
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
University Medical Center Ljubljana Ljubljana Slovenia
Rigshospitalet Copenhagen Denmark
Pirkanmaan hyvinvointialue Tampere Finland
Centre Hospitalier Lyon Sud Pierre Benite France
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Children’s Health Ireland Dublin Ireland
Cjeydplgp Uryaxewyrypxui Syuvfysqz Woluwe-Saint-Lambert Belgium
Udspgjmtpz Mnzuezf Cmbfkw Hszcimkgbpguprzob Hamburg Germany
Uoxoyafvirrhqvjfckjgr Eqpte Acr Essen Germany
Uaeazjlwljwcfrkgbjstr Aaojqizw Augsburg Germany
Fpwhfpgl nynwrncrl Mmzty a Hqscujb Prague Czechia
Apaxbualti Ptxcdtdt Hejwfjrz Dg Mxyilycue Marseille France
Hqrtdven Uhrzjsxbnu Cwpynzi Hxcejtuz Helsinki Finland
Ajcxih Ulmiibxbva Hcztsncg Aarhus Denmark
Adhjtow Onseallmaiq Uxunhdlvjlvcd Cpqsmlfunhjb Dxirp Sepklf E Dovvi Sbxcghc Dh Tnztuy Turin Italy
Uwcntifofswskcbadorgw Wqpahmuua Avs Wuerzburg Germany
Fsluxnlsh Piwy Lc Ikirafovfoozz Bzlusjolz Dcq Hoduplhl Uoypdjbxaspyq Li Pgs Madrid Spain
Hxlbmxwc Vdqn dvaienbv Barcelona Spain
Ggwnvg Ugkrcwfbep Flqucmtsz Frankfurt Germany
Cqmutw Ojwmc Lhcheql Lille France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
31.10.2022
Belgium Belgium
Not recruiting
31.10.2022
Czechia Czechia
Not recruiting
31.10.2022
Denmark Denmark
Not recruiting
31.10.2022
Finland Finland
Not recruiting
31.10.2022
France France
Not recruiting
31.10.2022
Germany Germany
Not recruiting
31.10.2022
Greece Greece
Not recruiting
31.10.2022
Hungary Hungary
Not recruiting
31.10.2022
Ireland Ireland
Not recruiting
31.10.2022
Italy Italy
Not recruiting
31.10.2022
Norway Norway
Not recruiting
31.10.2022
Slovenia Slovenia
Not recruiting
31.10.2022
Spain Spain
Not recruiting
31.10.2022
Sweden Sweden
Not recruiting
31.10.2022
The Netherlands The Netherlands
Not recruiting
31.10.2022

Trial locations

DAY101 (tovorafenib)
This medication is designed to target specific genetic changes (RAF alterations) in pediatric low-grade brain tumors called gliomas. It is taken as a monotherapy, meaning it is used alone without other cancer medications. It works by blocking certain proteins that contribute to tumor growth.

Standard of care chemotherapy
This refers to the currently accepted and commonly used chemotherapy treatments for pediatric low-grade gliomas. These are established cancer-fighting medications that have been proven effective and are considered the usual treatment approach for this type of brain tumor.

Investigated diseases:

Pediatric Low-Grade Glioma – A slow-growing brain tumor that typically develops in children and young adults, primarily affecting the brain or spinal cord tissue. It forms from glial cells, which are supportive cells in the central nervous system. When caused by RAF gene alterations, these tumors show abnormal cell growth patterns. The tumor can develop in various parts of the brain or spinal cord, leading to different symptoms depending on its location. These tumors tend to grow slowly over time, and their behavior can vary significantly between patients.

Trial ID:
2024-510742-13-00
Protocol code:
DAY101-002
NCT ID:
NCT05566795
Trial Phase:
Therapeutic confirmatory (Phase III)

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