Recruiting
Phase 2

DAY101

Sponsor:

Day One Biopharmaceuticals, Inc.

Code:

NCT04775485

Conditions

Low-grade Glioma

Advanced Solid Tumor

Eligibility Criteria

Sex: All

Age: 0 - 25

Healthy Volunteers: Not accepted

Interventions

Tovorafenib

Study Details

Brief 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.

Conditions

Low-grade Glioma

Advanced Solid Tumor

Study ID

NCT04775485

Start date

Apr 22, 2021

Status verified date

Mar, 2025

Completion date

May 31, 2027

Anticipated

Primary completion date

May 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 25

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Low Grade Glioma \& Low-Grade Glioma Extension: a relapsed or progressive LGG with documented known activating BRAF alteration.
  • Advanced Solid Tumor: locally advanced or metastatic solid tumor with documented known or expected to be activating RAF fusion.
  • Participants must have histopathologic verification of malignancy at either original diagnosis or relapse.
  • Must have received at least one line of prior systemic therapy and have documented evidence of radiographic progression.
  • Must have at least 1 measurable lesion as defined by RANO (Arms 1 \& 2) or RECIST v1.1 (Arm 3) criteria

Exclusion Criteria:

  • 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

Study Design

Enrollment

141 participants

Anticipated

Allocation

Non randomized

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Arm 1: Low-Grade Glioma

Participants with recurrent or progressive low-grade glioma will receive 420 milligrams/meters square (mg/m\^2) of tovorafenib weekly according to dose rounding guidelines and according to their baseline body surface area (BSA).

experimental: Arm 2: Low-Grade Glioma Expanded Access

Participants with recurrent or progressive low-grade glioma will receive 420 mg/m\^2 of tovorafenib weekly according to dose rounding guidelines and according to their baseline BSA.

experimental: Arm 3: Advanced Solid Tumor

Participants with advanced solid tumors will receive 420 mg/m\^2) of tovorafenib weekly according to dose rounding guidelines and according to their baseline BSA.

Interventions

Tovorafenib

Tovorafenib is an oral Type II RAF kinase inhibitor available in 100 mg immediate-release tablet or 25 mg/milliliter (mL) powder for reconstitution.

Primary outcome measure

  • Arm 1: Overall response rate [ Time Frame: Up to 48 months ]
  • Arm 2: Number of participants reporting adverse events [ Time Frame: Up to 48 months ]
  • Arm 2: Number of participants with clinically significant changes in clinical chemistry parameters [ Time Frame: Up to 48 months ]
  • Arm 2: Number of participants with clinically significant changes in hematology parameters [ Time Frame: Up to 48 months ]
  • Arm 3: Overall response rate [ Time Frame: Up to 48 months ]

Central Contacts and Locations

Central contacts

Day One Biopharmaceuticals, Inc.

650-484-0899firefly-1@dayonebio.com

Locations

UCSF Benioff Children's Hospital

Recruiting

San Francisco, California, United States, 94143

Children's National Medical Center

Recruiting

Washington, District of Columbia, United States, 20010

Contacts

braintumorresearch@childrensnational.org

202-476-5000

Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Dana-Farber Cancer Institute

Recruiting

Boston, Massachusetts, United States, 02215

CS Mott Children's Hospital

Recruiting

Ann Arbor, Michigan, United States, 48109

St. Louis Children's Hospital

Recruiting

Saint Louis, Missouri, United States, 63110

NYU Langone Health

Recruiting

New York, New York, United States, 10016

Duke Cancer Center

Recruiting

Durham, North Carolina, United States, 27710

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Texas Children's Hospital

Recruiting

Houston, Texas, United States, 77030

University of Utah

Recruiting

Salt Lake City, Utah, United States, 84113

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Centre Hospitalier Universitaire Ste-Justine

Recruiting

Montreal, Quebec, Canada, H3T 1C5

Montreal Children's Hospital

Recruiting

Montreal, Quebec, Canada, H4A 3J1

Centre Mère-Enfant Soleil du CHU

Recruiting

Québec, Quebec, Canada, G1V 4G2

More Information

Sponsor

Day One Biopharmaceuticals, Inc.

Last update posted

Apr 10, 2025

Last verified

Mar, 2025

Keywords

  • Low-grade Glioma
  • Advanced Solid Tumor
  • FIREFLY-1
  • DAY101
  • Tovorafenib

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Day One Biopharmaceuticals, Inc. on 2025-04-10.