MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
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|ClinicalTrials.gov Identifier: NCT05286788|
Recruitment Status : Not yet recruiting
First Posted : March 18, 2022
Last Update Posted : December 1, 2022
|Condition or disease||Intervention/treatment||Phase|
|Adamantinous Craniopharyngioma Recurrent Adamantinomatous Craniopharyngioma||Drug: Binimetinib Oral Tablet [Mektovi]||Phase 2|
Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating pediatric brain tumor that lacks medical anti-tumor therapies. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life and becomes more challenging and risky in the setting of recurrent disease. Recent discoveries regarding the biological characteristics of ACP indicate that available agents, including Mitogen-activated protein kinase/extracellular signal-regulated kinase (MAPK/ERK) pathway inhibitors may have efficacy in the control of ACP. Binimetinib is one such agent.
In this study, up to 38 patients will receive oral binimetinib at the recommended phase 2 pediatric dose (RP2D) of 32 mg/m2/dose PO every 12 hours for 4 weeks which represents one cycle. Cycles will last 28 days and treatment may continue for up to two years (26 cycles).
It will be a multi-center Phase 2 trial with two strata for patients aged >1 year and <25 years with measurable ACP who may have been previously treated with radiation (Stratum 1, 18 patients) or without radiation (Stratum 2, 18 patients).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||38 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||MEKTOVI® (binimetinib)|
|Masking:||None (Open Label)|
|Official Title:||Phase 2 Study of the MEK Inhibitor MEKTOVI® (Binimetinib) for the Treatment of Pediatric Adamantinomatous Craniopharyngioma|
|Estimated Study Start Date :||December 31, 2022|
|Estimated Primary Completion Date :||December 31, 2024|
|Estimated Study Completion Date :||December 31, 2026|
Experimental: Stratum 1 and Stratum 2
Stratum 1: Patients with progressive or recurrent adamantinomatous craniopharyngiomas following radiation therapy.
Stratum 2: Patients with measurable adamantinomatous craniopharyngioma who have undergone surgery but have not previously received radiation therapy. Progressive disease is allowed but not required
Drug: Binimetinib Oral Tablet [Mektovi]
Binimetinib oral continuous dosing 32 mg/m2 PO BID for 4 weeks
Other Name: MEKTOVI
- Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with oral binimetinib [ Time Frame: 24 months ]To calculate the number of patients who experience sustained objective response rate [minor response (MR) + partial response (PR) + complete response (CR)] of patients with recurrent/progressive previously irradiated Adamantinomatous Craniopharyngioma to treatment with oral binimetinib (Stratum 1).
- Sustained objective response rate of patients with measurable ACP who have undergone surgery but have not been previously treated with radiation to treatment with oral binimetinib [ Time Frame: 24 months ]To calculate the number of patients who experience sustained objective response rate (MR + PR + CR) of patients with measurable Adamantinomatous Craniopharyngioma who have undergone surgery but have not been previously treated with radiation to treatment with oral binimetinib (Stratum 2).
- Biological effects of binimetinib on ACP tumor tissue and cyst fluid. [ Time Frame: 24 months ]To measure concentrations of various chemokines including IL-6, IL-8, IL-10 and CXCL1in cyst fluid or tumor or blood of ACP patients treated with binimetinib. Comparisons will be made with known levels in the literature and among patient samples from within the study.
- Toxicities associated with tocilizumab in children with ACP [ Time Frame: 24 months ]To calculate the number of participants with, as well as frequency and severity of, binimetinib-related Adverse Events as assessed by CTCAE v5.0 in children with recurrent or refractory ACP
- PFS of ACP patients treated with binimetinib after radiation [ Time Frame: 12 months ]To assess one-year progression-free survival (PFS) rates for patients with ACP who are treated with binimetinib following progression after radiation (Stratum 1).
- PFS of ACP patients treated with binimetinib who have not received radiation [ Time Frame: 12 months ]To assess one-year progression-free survival (PFS) rates for patients with ACP who are treated with binimetinib who have not previously received radiation (Stratum 2).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05286788
|Contact: Leonie Mikaelfirstname.lastname@example.org|
|Contact: Dorothy Crabtree||16147228693||Dorothy.Crabtree@nationwidechildrens.org|
|Study Chair:||Kathleen H Dorris||Children's Hospital Colorado|
|Study Chair:||Todd C Hankinson||Children's Hospital Colorado|
|Principal Investigator:||Maryam Fouladi||Nationwide Children's Hospital|