Cure My Disease

Don't miss your cure!

 Log in / Sign up

New pill may replace chemo for kids with brain tumors

NCT ID NCT05566795

Disease control Sponsor: Day One Biopharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new drug called DAY101 (tovorafenib) against standard chemotherapy for children and young adults with a type of brain tumor called low-grade glioma that has a specific gene change (RAF alteration). The goal is to see if the new drug works better at shrinking tumors and delaying growth, while possibly having fewer side effects. About 418 participants under 25 years old who need their first treatment will be randomly assigned to receive either DAY101 pills or standard chemo.

Why investors are watching

Day One Biopharmaceuticals is running a Phase 3 trial of its drug tovorafenib against standard chemotherapy in children with a type of brain tumor called low-grade glioma. The trial is large for this small company, with 418 participants, and its outcome will largely determine whether tovorafenib becomes a first-line treatment for these children. Because Day One is small, this single trial's result carries outsized weight for the company's future.

If it works: If the trial shows tovorafenib controls the disease better than chemotherapy, the company could seek approval to sell the drug for this use. That would give Day One a commercial product in a defined pediatric patient group.

If it fails: If the trial fails or is delayed, Day One would have no approved product from this study and would face the cost and setback of a negative readout. Trials in cancer often fail, so this outcome is a real possibility.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

418 people

The number who actually took part.

Started

Feb 2023

Expected to finish

Jun 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 25 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Less than 25 years of age with LGG with known activating RAF alteration. * Histopathologic diagnosis of glioma or glioneuronal tumor. * At least one measurable lesion as defined by RANO criteria. * Meet indication for first-line systemic therapy. Exclusion Criteria: * Participant has any of the following tumor-histological findings: 1. Schwannoma 2. Subependymal giant cell astrocytoma (Tuberous Sclerosis) 3. Diffuse intrinsic pontine glioma, even if histologically diagnosed as World Health Organization (WHO) Grade I-II * Participant's tumor has additional pathogenic molecular alterations, including but not limited to a) isocitrate dehydrogenase (IDH) 1/2 mutation, b) Histone H3 mutation, and c) neurofibromatosis Type 1 (NF-1) loss of function alteration. * Known or suspected diagnosis of NF-1/ neurofibromatosis Type 2 (NF-2). * Prior or ongoing nonsurgical anticancer therapy for this indication (eg, chemotherapy, oral/IV targeted therapy) including radiation.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Low grade glioma are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

childhood low-grade glioma low grade glioma Noonan syndrome 5

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Low grade glioma Pediatric low-grade glioma Rapidly accelerated fibrosarcoma (RAF) altered glioma

Contacts and locations

Locations

  • Aarhus Universitetshospital

    Aarhus, Central Jutland, 8200, Denmark

  • Aghia Sofia General Children's Hospital

    Athens, 11527, Greece

  • Albany Medical Center

    Albany, New York, 12208, United States

  • Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8, Canada

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, England, L12 2AP, United Kingdom

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arnold Palmer Hospital for Children

    Orlando, Florida, 32806, United States

  • Astrid Lindgrens Barnsjukhus Solna

    Stockholm, 171 77, Sweden

  • Athens General Children's Hospital

    Athens, 11527, Greece

  • Azienda Ospedale Università di Padova

    Padova, 35128, Italy

  • Azienda Ospedaliera U. Meyer

    Florence, 50139, Italy

  • Azienda Ospedaliera di Rilievo Nazional Santobono Pausilipon

    Naples, 80131, Italy

  • Azienda Sanitaria Universitaria Friuli Centrale - P.O. Santa Maria della Misericordia

    Udine, 33100, Italy

  • Azienda Universitaria Ospedaliera Consorziale - Policlinico di Bari

    Bari, 70124, Italy

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, England, B4 6NH, United Kingdom

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3N1, Canada

  • CCHE

    Cairo, Cairo Governorate, 11441, Egypt

  • CHU Sainte-Justine

    Montreal, Quebec, H3T IC5, Canada

  • Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ, United Kingdom

  • Centre Hospitalier Universitaire Vaudois Lausanne

    Lausanne, 1011, Switzerland

  • Centre Hospitalier de l'Université Laval et Centre Mère-Enfant Soleil

    Québec, G1V 4G2, Canada

  • Centre Léon Bérard

    Lyon, 69008, France

  • Centre de Lutte contre le Cancer - Centre Oscar Lambret

    Lille, 59020, France

  • Chang Gung Memorial Hospital - Linkou Branch

    Taoyuan, 333, Taiwan

  • Charité Universitätsmedizin Berlin

    Berlin, 13353, Germany

  • Children's Health Ireland at Crumlin

    Crumlin, Dublin, D12 N512, Ireland

  • Children's Health Queensland Hospital and Health Service

    South Brisbane, Queensland, 4101, Australia

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital London Health Sciences Centre

    London, Ontario, N6A 5W9, Canada

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital at Westmead

    Westmead, 2145, Australia

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's Hospital of Orange County Main Campus - Orange

    Orange, California, 92868, United States

  • Children's Minnesota

    Minneapolis, Minnesota, 55404, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Children's of Alabama

    Birmingham, Alabama, 35233, United States

  • Childrens MS Cncr Ctr Bld Dsr

    Jackson, Mississippi, 39216, United States

  • Cleveland Clinic Main Campus

    Cleveland, Ohio, 44195, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, 1200, Belgium

  • Columbia University

    New York, New York, 10032, United States

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106-3322, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Dell Children's Medical Center of Central Texas

    Austin, Texas, 78723, United States

  • Drottning Silvias barn- och ungdomssjukhus

    Gothenburg, 416 85, Sweden

  • Duke Cancer Institute

    Durham, North Carolina, 27705, United States

  • Evangelische Klinikum Bethel (EvKB)

    Bielefeld, 33617, Germany

  • Fakultní Nemocnice Brno - D?tská Nemocnice

    Brno, 625 00, Czechia

  • Fondazione IRCCS - Istituto Nazionale dei Tumori

    Milan, Milan, 20133, Italy

  • Great Ormond Street Hospital for Children

    Oxford, England, WC1N 3JH, United Kingdom

  • Grupo de Apoio ao Adolescente e à Criança com Câncer (GRAACC)

    São Paulo, 04039-001, Brazil

  • Gustave Roussy

    Villejuif, 94805, France

  • Helsingin yliopistollinen sairaala (HUS)

    Helsinki, FI-00290, Finland

  • Hospital Infantil Universitario Niño Jesús

    Madrid, 28009, Spain

  • Hospital Sant Joan de Déu Barcelona

    Barcelona, 08950, Spain

  • Hospital Universitari Vall d'Hebrón

    Barcelona, 08035, Spain

  • Hospital Universitari i Politècnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario Cruces

    Barakaldo, 48903, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Universitario Virgen del Rocío

    Seville, 41013, Spain

  • Hôpital de la Timone

    Marseille, 13005, France

  • Institut Curie

    Paris, 75248, France

  • Istituto Giannina Gaslini

    Genova, 16147, Italy

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701, United States

  • King Hussein Cancer Foundation - King Hussein Cancer Center

    Amman, Muhafazat al-Asima, 11941, Jordan

  • Leeds Teaching Hospital NHS Trust

    Leeds, England, LS1 3EX, United Kingdom

  • Maine Children's Cancer Program

    Scarborough, Maine, 04074, United States

  • Manchester University NHS Foundation Trust

    Manchester, England, M13 PWL, United Kingdom

  • McMaster Children's Hospital

    Hamilton, Ontario, L8S 4L8, Canada

  • Medizinische Hochschule Hannover

    Hanover, Lower Saxony, 30625, Germany

  • Medizinische Universität Innsbruck

    Innsbruck, Tyrol, 6020, Austria

  • Medizinische Universität Wien

    Vienna, 1090, Austria

  • Motol University Hospital

    Prague, 5 150 06, Czechia

  • NHS Greater Glasgow and Clyde

    Glasgow, Scotland, G51 4TF, United Kingdom

  • New York University Langone Health

    New York, New York, 10016, United States

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

  • Norton Children's Hospital

    Louisville, Kentucky, 40202, United States

  • Oregon Health Science University

    Portland, Oregon, 97239, United States

  • Oslo Universitetssykehus

    Oslo, 372, Norway

  • Ospedale Infantile Regina Margherita

    Torino, Turin, 10126, Italy

  • Ospedale Pediatrico Bambino Gesù

    Roma, Rome, 00165, Italy

  • Packard Children's Hospital Stanford

    Palo Alto, California, 94304, United States

  • Perth Children's Hospital

    Nedlands, 6009, Australia

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Prinses Maxima Centrum Kinderoncologie

    Utrecht, 3584 CS, Netherlands

  • Rigshospitalet

    Copenhagen, 2100, Denmark

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana, 46202, United States

  • Royal Belfast Hospital for Sick Children

    Belfast, Northern Ireland, BT9 7AB, United Kingdom

  • Schneider Children's Medical Center of Israel

    Petah Tikva, 49202, Israel

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Semmelweis Egyetem Tűzoltó utcai II. Sz. Gyermekgyógyászati Kliniká

    Budapest, 1094, Hungary

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Severance Hospital

    Seoul, 03722, South Korea

  • Sheffield Children's Hospital

    Sheffield, South Yorkshire, S10 2TH, United Kingdom

  • SickKids - The Hospital for Sick Children

    Toronto, M5G 1X8, Canada

  • Sidra Medicine

    Doha, 26999, Qatar

  • SingHealth Group - KK Women's and Children's Hospital

    Singapore, 229899, Singapore

  • Skånes Universitetssjukhus - Lund

    Lund, 221 85, Sweden

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Starship Paediatric Blood & Cancer Center

    Grafton, 1023, New Zealand

  • Stollery Children's Hospital

    Edmonton, Alberta, T6G 2B7, Canada

  • Sydney Children's Hospital - Randwick

    Randwick, 2031, Australia

  • Taipei Medical University Hospital

    Taipei, 110, Taiwan

  • Tampereen Yliopistollinen Sairaala

    Tampere, 33520, Finland

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • The Christie Hospital

    Manchester, M20 4BX, United Kingdom

  • The Edmond and Lily Safra Children's Hospital

    Ramat Gan, 5265601, Israel

  • The Montreal Children's Hospital

    Montreal, Quebec, H4A 3J1, Canada

  • The Newcastle Upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, England, NE1 4LP, United Kingdom

  • The Royal Children's Hospital - Children's Cancer Centre

    Parkville, 3052, Australia

  • The Royal Marsden NHS Foundation Trust

    Sutton, Surrey, SM2 5PT, United Kingdom

  • UCSF Benioff Children's Hospital

    San Francisco, California, 94158, United States

  • Universitaets - Kinderspital Zürich

    Zurich, 8032, Switzerland

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, 45147, Germany

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • Universitair Ziekenhuis Leuven - Campus Gasthuisberg

    Leuven, 3000, Belgium

  • Universitetssykehuset Nord-Norge - Tromsø

    Tromsø, Troms, 9038, Norway

  • University Hospital Southampton NHS Foundation Trust

    Southampton, Hampshire, SO16 6YD, United Kingdom

  • University Hospitals Bristol and Weston NHS Foundation Trust

    Bristol, BS2 8HW, United Kingdom

  • University of Chicago

    Chicago, Illinois, 60637, United States

  • University of Florida Health

    Gainesville, Florida, 32610, United States

  • University of Iowa Hospitals Clinics

    Iowa City, Iowa, 52242, United States

  • University of Michigan - - C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198-6878, United States

  • University of Rochester

    Rochester, New York, 14642, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • University of Wisconsin - Madison

    Madison, Wisconsin, 53792, United States

  • Universitätsklinikum Augsburg

    Augsburg, Bavaria, 86156, Germany

  • Universitätsklinikum Erlangen

    Erlangen, Bavaria, 91054, Germany

  • Universitätsklinikum Frankfurt

    Frankfurt am Main, Hesse, 60590, Germany

  • Universitätsklinikum Freiburg

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • Universitätsklinikum Heidelberg

    Heidelberg, 69120, Germany

  • Universitätsklinikum Leipzig

    Leipzig, Saxony, 4103, Germany

  • Universitätsklinikum Tübingen

    Tübingen, 72076, Germany

  • Universitätsklinikum Würzburg

    Würzburg, Bavaria, 97080, Germany

  • Univerzitetni Klinini Center Ljubljana

    Ljubljana, 1000, Slovenia

  • Women's and Children's Health Network

    North Adelaide, 5006, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.