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NCT04775485PHASE2Recruiting

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

Day One Biopharmaceuticals, Inc.

Start Date

4/22/2021

Completion Date

5/31/2027

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

Age Range: No minimum to 25 years

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

Interventions

DRUG

Tovorafenib

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Conditions

Low-grade GliomaAdvanced Solid Tumor

Locations

UCSF Benioff Children's Hospital

San Francisco, California 94143

United States

Children's National Medical Center

Washington D.C., District of Columbia 20010

United States

Lurie Children's Hospital of Chicago

Chicago, Illinois 60611

United States

Johns Hopkins Hospital

Baltimore, Maryland 21231

United States

Dana-Farber Cancer Institute

Boston, Massachusetts 02215

United States

CS Mott Children's Hospital

Ann Arbor, Michigan 48109

United States

St. Louis Children's Hospital

St Louis, Missouri 63110

United States

NYU Langone Health

New York, New York 10016

United States

Duke Cancer Center

Durham, North Carolina 27710

United States

Doernbecher Children's Hospital Oregon & Health Science University

Portland, Oregon 97239

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Texas Children's Hospital

Houston, Texas 77030

United States

University of Utah

Salt Lake City, Utah 84113

United States

Seattle Children's Hospital

Seattle, Washington 98105

United States

Queensland Children's Hospital

Brisbane, 4101

Australia

Royal Children's Hospital

Parkville, 3052

Australia

Perth Children's Hospital

Perth, WA 6009

Australia

Sydney Children's Hospital

Randwick, NSW 2031

Australia

The Children's Hospital at Westmead

Westmead, 2145

Australia

Centre Hospitalier Universitaire Ste-Justine

Montreal, Quebec H3T 1C5

Canada

Montreal Children's Hospital

Montreal, Quebec H4A 3J1

Canada

Centre Mère-Enfant Soleil du CHU

Québec, Quebec G1V 4G2

Canada

Rigshospitalet

Copenhagen,

Denmark

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

Berlin, 13353

Germany

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

Heidelberg, 69120

Germany

Rambam Health Care Campus

Haifa, 3109601

Israel

Schneider Children's Medical Center of Israel

Petah Tikva, 4920235

Israel

The Chaim Sheba Medical Center

Ramat Gan, 5265601

Israel

Princess Maxima Center for Pediatric Oncology

Utrecht, 3584 CS

Netherlands

KK Women's and Children's Hospital

Singapore, 229899

Singapore

Seoul National University Hospital

Seoul, 3080

South Korea

Severance Hospital - Yonsei University

Seoul, 3722

South Korea

Universitäts-Kinderspital Zürich - Eleonorenstiftung

Zurich, 8032

Switzerland

UCL Great Ormond Street Institute of Child Health

London, WC1N 1EH

United Kingdom

Newcastle University

Newcastle upon Tyne, NE1 7RU

United Kingdom