New drug targets genetic weakness in tough cancers

NCT ID NCT04985604

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

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

Summary

This phase 2 trial tested the drug tovorafenib (DAY101) in 23 people aged 12 and older with recurrent or progressive solid tumors that have specific genetic changes in the MAPK pathway. The goal was to see how many patients' tumors shrank or disappeared. The study was terminated early, so the full results are not available.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Tovorafenib (also called DAY101), a targeted cancer drug taken as a tablet by mouth.
What this could lead to: If successful, this drug could offer a new treatment option for certain hard-to-treat cancers with specific genetic changes.
What could go wrong: This was a small, early-phase trial that was terminated, so results are limited. The drug may not work for all tumor types and can cause side effects.

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

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Conditions

The condition(s) this trial relates to.

astrocytoma (excluding glioblastoma) bladder transitional cell carcinoma colorectal cancer colorectal neoplasm familial pancreatic carcinoma lung adenocarcinoma melanoma neoplasm non-small cell lung carcinoma pancreatic neoplasm pilocytic astrocytoma spitzoid melanoma Thyroid Cancer, Papillary thyroid gland papillary carcinoma urinary bladder carcinoma Urinary Bladder Neoplasms

As listed by the trial registrant

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

BLADDER CANCER BLADDER UROTHELIAL CARCINOMA BRAF FUSION BRAF GENE FUSION COLORECTAL CANCER CRAF FUSION CRAF GENE AMPLIFICATION CRAF GENE FUSION MAP KINASE FAMILY GENE MUTATION MELANOMA NON-SMALL CELL ADENOCARCINOMA NON-SMALL CELL LUNG CANCER PANCREATIC ACINAR CARCINOMA PILOCYTIC ASTROCYTOMA PILOCYTIC ASTROCYTOMA, ADULT RAF MUTATION RAF1 FUSION RAF1 GENE AMPLIFICATION RAF1 GENE FUSION SOLID TUMOR SPITZOID MALIGNANT MELANOMA SPITZOID MELANOMA THYROID CANCER, PAPILLARY

Contacts and locations

Locations

Antwerp University Hospital

Edegem, Belgium
Asan Medical Center

Seoul, South Korea
Cancer Specialists of North Florida

Jacksonville, Florida, 32256, United States
Community North Cancer Center

Indianapolis, Indiana, 46250, United States
Dong-A University Hospital

Busan, South Korea
Hoag Health

Newport Beach, California, 92663, United States
Hopital de La Timone - APHM

Marseille, Bouches-du-Rhône, France
Hospital Clinic Barcelona

Barcelona, Spain
Hospital Universitari Vall d'Hebron

Barcelona, Spain
Hospital Universitario Ramón y Cajal

Madrid, Spain
Monash Medical Centre

Clayton, Victoria, Australia
OHSU Knight Cancer Institute

Portland, Oregon, 97239, United States
Princess Margaret Cancer Centre

Toronto, Ontario, Canada
Samsung Medical Center

Seoul, South Korea
Severance Hospital, Yonsei University Health System

Seoul, South Korea
The Angeles Clinic

Los Angeles, California, 90025, United States
The Hospital for Sick Children

Toronto, Ontario, Canada
UPMC Hillman Cancer Center

Pittsburgh, Pennsylvania, 15213, United States
University of Colorado Hospital

Aurora, Colorado, 80045, United States
Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, 37232, United States

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