A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors
Purpose
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
Eligibility
- Eligible Ages
- Between 6 Months and 25 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
Recruiting Locations
Children's National and nearby locations
Children's National Medical Center
Washington, District of Columbia 20010
Washington, District of Columbia 20010
Contact:
braintumorresearch@childrensnational.org
202-476-5000
braintumorresearch@childrensnational.org
202-476-5000
More Details
- NCT ID
- NCT04775485
- Status
- Recruiting
- Sponsor
- Day One Biopharmaceuticals, Inc.