A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

Last updated: March 27, 2025
Sponsor: Day One Biopharmaceuticals, Inc.
Overall Status: Active - Recruiting

Phase

2

Condition

Astrocytoma

Brain Tumor

Neurofibromatosis

Treatment

DAY101

Tovorafenib

Clinical Study ID

NCT04775485
DAY101-001/PNOC026
  • Ages 6-25
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Low Grade Glioma & Low-Grade Glioma Extension: a relapsed or progressive LGG withdocumented known activating BRAF alteration.

  • Advanced Solid Tumor: locally advanced or metastatic solid tumor with documentedknown or expected to be activating RAF fusion.

  • Participants must have histopathologic verification of malignancy at either originaldiagnosis or relapse.

  • Must have received at least one line of prior systemic therapy and have documentedevidence of radiographic progression.

  • Must have at least 1 measurable lesion as defined by RANO (Arms 1 & 2) or RECISTv1.1 (Arm 3) criteria

Exclusion

Exclusion Criteria:

  • Participant's tumor has additional previously-known activating molecularalterations.

  • Participant has symptoms of without radiographically recurrent or radiographicallyprogressive disease.

  • Known or suspected diagnosis of neurofibromatosis type 1 (NF-1) via genetic testingor current diagnostic criteria.

Other inclusion/exclusion criteria as stipulated by protocol may apply

Study Design

Total Participants: 141
Treatment Group(s): 2
Primary Treatment: DAY101
Phase: 2
Study Start date:
April 22, 2021
Estimated Completion Date:
May 31, 2027

Connect with a study center

  • Queensland Children's Hospital

    Brisbane, 4101
    Australia

    Active - Recruiting

  • Royal Children's Hospital

    Parkville, 3052
    Australia

    Active - Recruiting

  • Perth Children's Hospital

    Perth, WA 6009
    Australia

    Active - Recruiting

  • Sydney Children's Hospital

    Randwick, NSW 2031
    Australia

    Active - Recruiting

  • The Children's Hospital at Westmead

    Westmead, 2145
    Australia

    Active - Recruiting

  • Centre Hospitalier Universitaire Ste-Justine

    Montreal, Quebec H3T 1C5
    Canada

    Active - Recruiting

  • Montreal Children's Hospital

    Montreal, Quebec H4A 3J1
    Canada

    Active - Recruiting

  • Centre Mère-Enfant Soleil du CHU

    Québec, Quebec G1V 4G2
    Canada

    Active - Recruiting

  • Centre Hospitalier Universitaire Ste-Justine

    Montréal, QC H3T 1C5
    Canada

    Site Not Available

  • Rigshospitalet

    Copenhagen,
    Denmark

    Active - Recruiting

  • Charité Universitätsmedizin Berlin, Campus Virchow Klinikum, Otto-Heubner-Centrum für Kinder

    Berlin, 13353
    Germany

    Active - Recruiting

  • Hopp-Kindertumorzentrum Heidelberg (KiTZ), KiTZ Clinical Trial Unit (ZIPO)

    Heidelberg, 69120
    Germany

    Active - Recruiting

  • Rambam Health Care Campus

    Haifa, 3109601
    Israel

    Active - Recruiting

  • Schneider Children's Medical Center of Israel

    Petah Tikva, 4920235
    Israel

    Active - Recruiting

  • The Chaim Sheba Medical Center

    Ramat Gan, 5265601
    Israel

    Active - Recruiting

  • Seoul National University Hospital

    Seoul, 3080
    Korea, Republic of

    Active - Recruiting

  • Severance Hospital - Yonsei University

    Seoul, 3722
    Korea, Republic of

    Active - Recruiting

  • Princess Maxima Center for Pediatric Oncology

    Utrecht, 3584 CS
    Netherlands

    Active - Recruiting

  • KK Women's and Children's Hospital

    Singapore, 229899
    Singapore

    Active - Recruiting

  • Universitäts-Kinderspital Zürich - Eleonorenstiftung

    Zürich, 8032
    Switzerland

    Active - Recruiting

  • UCL Great Ormond Street Institute of Child Health

    London, WC1N 1EH
    United Kingdom

    Active - Recruiting

  • Newcastle University

    Newcastle Upon Tyne, NE1 7RU
    United Kingdom

    Active - Recruiting

  • UCSF Benioff Children's Hospital

    San Francisco, California 94143
    United States

    Active - Recruiting

  • Children's National Medical Center

    Washington, District of Columbia 20010
    United States

    Active - Recruiting

  • Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Johns Hopkins Hospital

    Baltimore, Maryland 21231
    United States

    Terminated

  • Dana-Farber Cancer Institute

    Boston, Massachusetts 02215
    United States

    Active - Recruiting

  • CS Mott Children's Hospital

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • St. Louis Children's Hospital

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • NYU Langone Health

    New York, New York 10016
    United States

    Active - Recruiting

  • Duke Cancer Center

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • Doernbecher Children's Hospital Oregon & Health Science University

    Portland, Oregon 97239
    United States

    Terminated

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Texas Children's Hospital

    Houston, Texas 77030
    United States

    Active - Recruiting

  • University of Utah

    Salt Lake City, Utah 84113
    United States

    Active - Recruiting

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Active - Recruiting

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