BridgeMD
New treatment option for Craniopharyngioma, Child
Official title Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults
ClinicalTrials.gov ID: NCT05465174
What this study is testing
What is Tovorafenib?
Tovorafenib is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for craniopharyngioma, child.
Also referred to as DAY101, TAK580.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The current study assesses the tolerability and efficacy of monotherapy with pan-RAF-kinase (Tovorafenib) inhibition for the treatment of children and young adults with craniopharyngioma.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 39
You may be able to join if
- Newly Diagnosed Participants:
- Newly diagnosed craniopharyngioma, as based on imaging characteristics and central radiology review. Participants will initially be screened within...
- Participants must be surgical candidates for biopsy or resection and planned for standard of care biopsy or resection. Recurrent Participants:
- Recurrent craniopharyngioma, as based on histologic confirmation at time of initial diagnosis (participants with Adamantinomatous craniopharyngioma...
- Recurrent craniopharyngioma without prior histologic confirmation will initially be screened within confines of a screening consent and only those...
You likely can't join if
- Newly Diagnosed Participants: \- Participants should not have undergone any previous tumor-directed therapy. Recurrent Participants:
- Participants who have had chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those...
- Participants must be at least 1 week since the completion of therapy with a biologic or small molecule agent. For any agent with known adverse events...
- Participants should not have previously received any RAS-pathway, but have not received Tovorafenib will be eligible. All Participants:
- Rapidly progressive symptoms that require urgent surgery or radiation therapy, which would prevent central review and or preclude participation with...
- Uncontrolled symptoms of neuroendocrine dysfunction such as diabetes insipidus, hypothyroidism, panhypopituitarism (participants can be on...
See the full eligibility criteria
Who can join
- Newly Diagnosed Participants:
- Newly diagnosed craniopharyngioma, as based on imaging characteristics and central radiology review. Participants will initially be screened within confines of a screening consent and only those participants with...
- Participants must be surgical candidates for biopsy or resection and planned for standard of care biopsy or resection. Recurrent Participants:
- Recurrent craniopharyngioma, as based on histologic confirmation at time of initial diagnosis (participants with Adamantinomatous craniopharyngioma (ACP) will only be eligible for the recurrent arm).
- Recurrent craniopharyngioma without prior histologic confirmation will initially be screened within confines of a screening consent and only those participants with findings consistent with craniopharyngioma and without...
- Participants should be surgical candidates for biopsy or resection. If participants are not surgical candidates, but have available archival tumor tissue, they will be enrolled into the exploratory cohort.
- Participants must be willing to provide archival tissue, a minimum of 10-20 paraffin embedded unstained slides OR 1 block with tumor content of 40% or greater is required. Participants who do not meet this criteria may...
- Participants can have been previously treated with surgical resection alone, cyst drainage and biopsy alone, radiation therapy, other systemic therapies, or any combination thereof.
- Prior Therapy:
- Had their last dose of myelosuppressive chemotherapy \>= 21 days prior to study registration (\>=42 days if nitrosourea therapy).
- Had their last dose of hematopoietic growth factor \>=14 days (long-acting growth factor) or \>=7 days (short-acting growth factor) prior to study registration, or beyond the time during which adverse events (AEs) are...
- Had their last dose of biologic (anti-neoplastic agent) \>=7 days prior to study registration, or beyond the time during which AEs are known to occur.
- Had their last dose of monoclonal antibodies \>=21 days prior to study registration. Radiation:
- Had their last fraction of local irradiation to primary tumor \>=12 weeks prior to registration; investigators are reminded to review potentially eligible cases to avoid confusion with pseudo-progression.
- At least 14 days after local palliative radiation (small-port). All Participants:
- Age 1 to 39 years.
- Participants continuing on maintenance therapy after standard of care biopsy/resection must have measurable disease, as defined as lesions that can be accurately measured in two dimensions (longest diameter to be...
- Performance Score: Karnofsky \>= 50 for participants \> 16 years of age and Lansky \>= 50 for participants \<= 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair...
- Corticosteroids: Participants who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration. The participant steroid dose should be no more than a steroid-equivalent of...
- Organ Function Requirements:
- Adequate Bone Marrow Function defined as:
- Peripheral absolute neutrophil count (ANC) \>=1000/mm3.
- Platelet count \>= 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).
- Adequate Renal Function defined as- ---A serum creatinine \< 1.5 Upper Limit normal (ULN) based on age and gender.
- Adequate Liver Function defined as-
- Bilirubin (sum of conjugated + unconjugated) \<= 1.5 x upper limit of normal (ULN) for age (except in participants with documented Gilbert syndrome).
- Serum glutamic-pyruvic transaminase (SGPT)((alanine aminotransferase (ALT)) \<= 3 x ULN.
- Serum albumin \>=2 g/dL (20g/L).
- Adequate Neurologic Function defined as participants with seizure disorder may be enrolled if well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug.
- Adequate Pulmonary Function defined as no evidence of dyspnea at rest, no exercise intolerance due to pulmonary insufficiency, and a pulse oximetry of \> 92% while breathing room air.
- prothrombin time (PT) /partial thromboplastin time (PTT)/International Normalized Ratio (INR) within institutional normal limits or deemed appropriate for surgical intervention by the treating team for patients...
- The effects of Tovorafenib on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (non-hormonal contraception; barrier method of...
- A legal parent/guardian or participants must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.
- Ability to complete the PedsQL Core Module.
- Patients must enroll on PNOC COMP if PNOC COMP is open to accrual at the enrolling institution.
What rules you out
- Newly Diagnosed Participants: \- Participants should not have undergone any previous tumor-directed therapy. Recurrent Participants:
- Participants who have had chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from acute adverse events due to agents...
- Participants must be at least 1 week since the completion of therapy with a biologic or small molecule agent. For any agent with known adverse events that can occur beyond 1 week after administration, the period prior...
- Participants should not have previously received any RAS-pathway, but have not received Tovorafenib will be eligible. All Participants:
- Rapidly progressive symptoms that require urgent surgery or radiation therapy, which would prevent central review and or preclude participation with tumor-directed medical management alone.
- Uncontrolled symptoms of neuroendocrine dysfunction such as diabetes insipidus, hypothyroidism, panhypopituitarism (participants can be on supplemental medications for hormonal repletion; however, should be on...
- Clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to registration, ongoing cardiomyopathy, or current prolonged...
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to tovorafenib.
- Nausea and vomiting \>= Grade 2, malabsorption requiring supplementation, or significant bowel or stomach resection that would preclude adequate absorption.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.
- Participants who are receiving any other investigational agents.
- Women of childbearing potential must not be pregnant or breast-feeding.
- Current treatment with a strong cytochrome P4502C8(CYP2C8) inhibitor or inducer other than those allowed per Section 5.6.1. Medications that are substrates of CYP2C8 are allowed but should be used with caution.
- Participants with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- San Diego, California, United States
- San Francisco, California, United States
- Gainesville, Florida, United States
- Chicago, Illinois, United States
- Indianapolis, Indiana, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- Minneapolis, Minnesota, United States
- St Louis, Missouri, United States
- New York, New York, United States
- Durham, North Carolina, United States
- Philadelphia, Pennsylvania, United States
- Salt Lake City, Utah, United States
- New Lambton Heights, New South Wales, Australia
- Hobart, Tasmania, Australia
- Clayton, VIC 3168, Australia
- Parkville, Victoria, Australia
- Nedlands, Western Australia, Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 39 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; San Diego, California, United States; San Francisco, California, United States; Gainesville, Florida, United States; Chicago, Illinois, United States; Indianapolis, Indiana, United States and 14 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.