Low-grade GliomaNCT05760586
Expanded Access Program (EAP) for Tovorafenib (DAY101) in RAF-Altered, Relapsed or Refractory Low-Grade Glioma
A study testing Tovorafenib in people with Low-grade Glioma, run by Day One Biopharmaceuticals, Inc..
Trial summary
The DAY101-EAP is a multicenter, open-label, expanded access treatment protocol designed to provide access to tovorafenib (DAY101) for eligible patients. Read more about this study
- Who is it for?
- Ages 6 months to 25 years
- Conditions the study is for
- Low-grade Glioma
- What is being tested?
- Tovorafenib
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 25 years
- All sexes
- Child, Adult
Who may be able to join
- Patients must meet all of the following criteria to be eligible for enrollment in the EAP:
- Patients must be aged 6 months to 25 years, inclusive, with a relapsed or refractory low-grade glioma with a documented known or expected to be activating BRAF mutation or RAF fusion, as identified through molecular assays as routinely performed at CLIA-certified or other similarly certified laboratories.
- Patients must have histopathologic diagnosis of glioma or glioneuronal tumor (Grade 1 or 2 according to WHO classification for CNS tumors at either original diagnosis or relapse.
- Patients must have received at least one line of prior systemic therapy and have documented evidence of radiographic progression.
- Previous chemotherapy and/or targeted/immunotherapy/monoclonal antibody therapy must be completed at least 4 weeks or 5 half-lives (whichever is shorter) prior to the initiation of therapy.
- Chronic toxicities from prior anticancer therapy must be stable and at CTCAE Version 5.0 Grade ≤ 2.
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- Patients must have fully recovered from any prior surgery.
- Patients must have adequate hematologic function, as defined by the following:
- Absolute neutrophil count≥ 1000/mm3
- Platelet count≥ 75.0 × 109/L
- Hemoglobin≥ 10.0 g/dL (transfusions allowed up to 14 days prior to enrollment per institutional guidelines)
- Patients must have adequate hepatic and renal function, defined by the following:
- Total bilirubin ≤ 1.5 × upper limit of normal (ULN) for age (If patient has documented Gilbert's Disease, patient may be enrolled with Sponsor approval, provided that bilirubin is < 2.0 × ULN)
- Serum glutamic-pyruvic transaminase (SGPT)/alanine aminotransferase (ALT) ≤ 3 × ULN
- Serum glutamic-oxaloacetic transaminase (SGOT)/aspartate transaminase (AST) ≤ 3 × ULN
- Serum creatinine within normal limits, or estimated glomerular filtration rate ≥ 60 mL/min/1.73 m2 based on local institutional practice for determination
- Thyroid function tests must be consistent with stable thyroid function.
- Patients must be able to comply with treatment, laboratory monitoring, and required clinic visits for the duration of EAP participation.
- Male and female patients with reproductive potential must be willing to use birth control methods per Appendix E of protocol.
- Patients must be able to swallow tablets or liquid or administer through gastric access via feeding tube(12Fr or greater)
- Parent/guardian of child or adolescent patient must have the ability to understand, agree to, and sign the EAP informed consent form (ICF) and applicable pediatric assent form before initiation of any treatment-related procedures; patient must have the ability to give assent, as applicable, at the time of parental/guardian consent.
Who cannot join
- Patients meeting any of the following criteria are to be excluded from EAP participation:
- Patient's tumor has an additional previously known or expected to be activating molecular alteration(s) (e.g., histone mutation, IDH1/2 mutations, FGFR mutations or fusions, MYBL alterations, neurofribromatosis type 1 (NF-1) somatic or germline mutations).
- Patient has known or suspected diagnosis of neurofibromatosis type 1 (NF-1) via genetic testing or current diagnostic criteria.
- Patient has history of any major disease, other than the primary malignancy under EAP, that might interfere with safe protocol participation.
- Patient has major surgery within 14 days (2 weeks) prior to C1D1 (does not include central venous access, cyst fenestration or cyst drainage, or ventriculoperitoneal shunt placement or revision).
- Patient has clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to C1D1, ongoing cardiomyopathy, or current prolonged QT interval corrected for heart rate by Fridericia's formula (QTcF) interval > 470 milliseconds based on triplicate electrocardiogram (ECG) average.
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- Patient has an active systemic bacterial, viral, or fungal infection.
- Patient has malabsorption requiring supplementation, or significant bowel or stomach resection that would preclude adequate absorption of tovorafenib (DAY101).
- Concomitant medications that are strong inhibitors or inducers of CYP2C8 within 14 days before initiation of therapy. Concomitant medications that are substrates of BCRP with a narrow therapeutic index within 14 days before initiation of therapy.
- Patient is pregnant or lactating, or plans to become pregnant in the immediate future.
- Patient has any clinically significant skin toxicity at screening, that in the opinion of the investigator would increase risk of severe skin toxicity when using investigational product.
- There are other unspecified reasons that, in the opinion of the HCP, make the patient unsuitable for enrollment.
- Patient has CTCAE v5.0 Grade ≥3, CPK elevation ( > 5x UL - 10 X ULN)
About this study
Official title: Expanded Access to the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients With RAF-Altered, Relapsed or Refractory Low-Grade Glioma
The DAY101-EAP is a multicenter, open-label, expanded access treatment protocol designed to provide access to tovorafenib (DAY101) for eligible patients.
- Conditions
- Low-grade Glioma
- Being tested
- Tovorafenib
- Keywords
- DAY101, Toverafenib, Glioma, BRAF Mutation, RAF Alteration, Type II BRAF Inhibitor, Neoplasms
- Medical subject headings
- Glioma, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TovorafenibDrug
Also called DAY101
Oral RAF inhibitor provided as an immediate-release tablet (100 mg) or powder for reconstitution (25 mg/mL)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Low-grade GliomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Day One Biopharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05760586
- Sponsor's protocol ID
- DAY101-EAP
- Status last verified
- Aug 2024
- First submitted
- 2 Feb 2023
- Last updated
- 12 Aug 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access to the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients With RAF-Altered, Relapsed or Refractory Low-Grade Glioma
- ClinicalTrials.gov ID
- NCT05760586
- Acronym / sponsor ID
- DAY101-EAP
- Conditions
- Low-grade Glioma
- Keywords
- DAY101, Toverafenib, Glioma, BRAF Mutation, RAF Alteration, Type II BRAF Inhibitor, Neoplasms
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Day One Biopharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 8 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The DAY101-EAP is a multicenter, open-label, expanded access treatment protocol designed to provide access to tovorafenib (DAY101) for eligible patients.
Study design
Who may qualify
- Ages
- 6 months to 25 years
- Sex
- All
- Age groups
- Child, Adult
Key inclusion criteria (21 listed)
- Patients must meet all of the following criteria to be eligible for enrollment in the EAP:
- Patients must be aged 6 months to 25 years, inclusive, with a relapsed or refractory low-grade glioma with a documented known or expected to be activating BRAF mutation or RAF fusion, as identified through molecular assays as routinely performed at CLIA-certified or other similarly certified laboratories.
- Patients must have histopathologic diagnosis of glioma or glioneuronal tumor (Grade 1 or 2 according to WHO classification for CNS tumors at either original diagnosis or relapse.
- Patients must have received at least one line of prior systemic therapy and have documented evidence of radiographic progression.
- Previous chemotherapy and/or targeted/immunotherapy/monoclonal antibody therapy must be completed at least 4 weeks or 5 half-lives (whichever is shorter) prior to the initiation of therapy.
Key exclusion criteria (13 listed)
- Patient's tumor has an additional previously known or expected to be activating molecular alteration(s) (e.g., histone mutation, IDH1/2 mutations, FGFR mutations or fusions, MYBL alterations, neurofribromatosis type 1 (NF-1) somatic or germline mutations).
- Patient has known or suspected diagnosis of neurofibromatosis type 1 (NF-1) via genetic testing or current diagnostic criteria.
- Patient has history of any major disease, other than the primary malignancy under EAP, that might interfere with safe protocol participation.
- Patient has major surgery within 14 days (2 weeks) prior to C1D1 (does not include central venous access, cyst fenestration or cyst drainage, or ventriculoperitoneal shunt placement or revision).
- Patient has clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to C1D1, ongoing cardiomyopathy, or current prolonged QT interval corrected for heart rate by Fridericia's formula (QTcF) interval > 470 milliseconds based on triplicate electrocardiogram (ECG) average.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TovorafenibAlso: DAY101Oral RAF inhibitor provided as an immediate-release tablet (100 mg) or powder for reconstitution (25 mg/mL) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Tovorafenib in people with Low-grade Glioma, run by Day One Biopharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2024.