400 Participants Needed

DAY101 vs. Chemotherapy for Pediatric Brain Cancer

Recruiting at 158 trial locations
DO
Overseen ByDay One Clinical Trials Information
Age: < 65
Sex: Any
Trial Phase: Phase 3
Sponsor: Day One Biopharmaceuticals, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines a new treatment called tovorafenib (also known as DAY101) for children and young adults with low-grade glioma, a specific type of brain tumor. Researchers aim to determine if tovorafenib is more effective and safer than standard chemotherapy treatments. Participants must have a confirmed diagnosis of this tumor type with an activating RAF alteration. This trial is for individuals who have not yet begun any systemic cancer treatment. As a Phase 3 trial, it represents the final step before FDA approval, offering participants an opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does exclude those who have had prior or ongoing nonsurgical anticancer therapy for this condition.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Previous studies have shown that tovorafenib is generally well-tolerated. Research indicates that children taking tovorafenib may experience slower growth, but the treatment remains safe. The FDA has approved tovorafenib for treating certain brain tumors in children, confirming its safety for those cases. This approval provides additional confidence in its safety.12345

Why do researchers think this study treatment might be promising for brain cancer?

Researchers are excited about Tovorafenib because it offers a new approach to treating pediatric brain cancer. Unlike traditional chemotherapy, which attacks rapidly dividing cells indiscriminately, Tovorafenib specifically targets a mutated protein called BRAF, which is involved in cell growth and division. This targeted mechanism may lead to fewer side effects and improved effectiveness. By honing in on the root cause of certain brain tumors, Tovorafenib has the potential to revolutionize treatment options and improve outcomes for young patients.

What evidence suggests that this trial's treatments could be effective for pediatric low-grade glioma?

Research shows that tovorafenib, a new medication, holds promise for treating a specific brain tumor in children called low-grade glioma (LGG) with certain genetic changes. In earlier studies, about 46% of children who took tovorafenib experienced tumor shrinkage within three months. The U.S. FDA has approved tovorafenib for treating some children with low-grade glioma, indicating confidence in its effectiveness. In this trial, participants will receive either tovorafenib or the investigator's choice of standard chemotherapy. These findings suggest that tovorafenib could be a promising treatment option for children with this type of brain cancer.12456

Are You a Good Fit for This Trial?

This trial is for children and young adults under 25 with a type of brain tumor called low-grade glioma that has a specific genetic change (RAF alteration). They should have a measurable tumor but can't have had any previous cancer treatments like chemo or radiation, and shouldn't have certain other medical conditions or additional genetic changes in their tumors.

Inclusion Criteria

I need treatment for my condition for the first time.
My diagnosis is a type of brain tumor called glioma or glioneuronal.
At least one measurable lesion as defined by RANO criteria
See 1 more

Exclusion Criteria

My tumor is identified as Schwannoma, SEGA, or DIPG.
I am currently undergoing or have completed non-surgical cancer treatment.
My tumor has other gene changes that make it grow.
See 1 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either tovorafenib monotherapy or standard of care chemotherapy. Treatment cycles repeat every 28 days until disease progression, unacceptable toxicity, or withdrawal.

Up to 60 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may continue therapy beyond progressive disease if deemed beneficial

Long-term

What Are the Treatments Tested in This Trial?

Interventions

  • Chemotherapeutic Agent
  • DAY101
Trial Overview The study compares DAY101, which is an experimental drug, to the standard chemotherapy drugs usually given for this condition. It's designed to see which treatment works better as the first line of attack against these brain tumors in kids and young adults.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Active Control
Group I: TovorafenibExperimental Treatment1 Intervention
Group II: Investigator's choice of Standard of care therapyActive Control1 Intervention

Chemotherapeutic Agent is already approved in United States for the following indications:

🇺🇸
Approved in United States as Ojemda for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Day One Biopharmaceuticals, Inc.

Lead Sponsor

Trials
8
Recruited
1,100+

SIOPe Brain Tumor Group LOGGIC Consortium

Collaborator

Trials
1
Recruited
400+

Published Research Related to This Trial

A two-year-old girl with a low-grade glioma and a BRAF-V600E mutation showed significant improvement and near-complete tumor resolution after being treated with the targeted therapy dabrafenib, following failure of standard chemotherapy.
This case suggests that children with low-grade gliomas harboring BRAF-V600E mutations may benefit more from targeted therapies like BRAF inhibitors rather than traditional chemotherapy, highlighting the need for further clinical trials to compare these treatment approaches.
Management of Inoperable Supra-Sellar Low-Grade Glioma With BRAF Mutation in Young Children.Howden, K., Chapman, S., Serletis, D., et al.[2023]
In a phase 2 trial involving 110 pediatric patients with low-grade glioma and BRAF V600 mutations, the combination of dabrafenib and trametinib resulted in a significantly higher overall response rate (47%) compared to standard chemotherapy (11%).
Dabrafenib plus trametinib also demonstrated a longer median progression-free survival (20.1 months) and a better safety profile, with fewer severe adverse events (47% vs. 94% for chemotherapy), making it a promising first-line treatment option.
Dabrafenib plus Trametinib in Pediatric Glioma with BRAF V600 Mutations.Bouffet, E., Hansford, JR., Garrè, ML., et al.[2023]
In a phase I trial involving 51 children with recurrent CNS tumors, lenalidomide was well-tolerated at doses up to 116 mg/m²/day, with myelosuppression being the primary side effect.
The trial showed some antitumor activity, with two objective responses in low-grade gliomas, indicating potential efficacy of lenalidomide in treating certain types of pediatric CNS tumors.
Phase I trial of lenalidomide in pediatric patients with recurrent, refractory, or progressive primary CNS tumors: Pediatric Brain Tumor Consortium study PBTC-018.Warren, KE., Goldman, S., Pollack, IF., et al.[2021]

Citations

The type II RAF inhibitor tovorafenib in relapsed/refractory ...These data indicate that tovorafenib could be an effective therapy for BRAF-altered, relapsed/refractory pLGG.
Tovorafenib Approved for Some Children with Low-Grade ...The study's investigators also noted that children's normal growth trajectory slowed during treatment with tovorafenib. Brain tumors, including ...
Tovorafenib for Relapsed or Refractory BRAF-altered ...The major efficacy outcome measure was radiologic overall response rate (ORR), defined as the proportion of patients with complete response, partial response, ...
Day One's OJEMDA™ (tovorafenib) Receives US FDA ...The US Food and Drug Administration (FDA) has approved OJEMDA (tovorafenib), a type II RAF inhibitor, for the treatment of patients 6 months of age and older ...
Clinical study results | OJEMDA™ (tovorafenib)ON SCREEN: 46% of children (18 out of 39) saw tumor shrinkage at 3 months after their first MRI scan* *These data were collected during the clinical study, and ...
6.ojemda.comojemda.com/
OJEMDA™ (tovorafenib): Pediatric low grade-glioma (pLGG ...OJEMDA is a prescription medicine used to treat certain types of brain tumors (cancers) called gliomas in patients 6 months and older.
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