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Also known as Momelotinib
Momelotinib treatment · 1 trial · 2 indications
The maximum tolerated dose (MTD) is defined by a target dose-limiting toxicities (DLT) rate of 30%, assessed during the observation window by a 3+3 design. The 3+3 design will enroll a first cohort of 3 patients at the starting dose of 200 mg/d: * If 0 of the 3 patients at the dose of 200 mg/d experienced a DLT during the first 4-week cycle of momelotinib, the dose will escalate to 300 mg/d for the next cohort. * If 1 of the 3 patients at the dose of 200 mg/d has a DLT, a cohort of three additional patients will be treated at the same dose of 200 mg/d. * If \> 1 patient of the 3 patients at the dose of 200 mg/d have a DLT, the dose will de-escalate to 150 mg/d for the next cohort. The same process will be repeated until reaching the MTD. In total, between 6 and 18 patients will be enrolled during this safety run-in phase.
Determination of overall clinical response rate at 24 weeks after momelotinib initiation on VEXAS related symptoms (including complete (CR) or partial response (PR)) : * CR includes complete disappearance of symptoms related to systemic inflammation according to treating physician and daily dose of steroids ≤ 10 mg/d (equivalent prednisolone). * PR includes complete disappearance of symptoms related to systemic inflammation according to treating physician and reduction of at least 50% of daily dose steroids compared to baseline and/or daily dose of steroids \> 10 mg/d.
| Arm | Type | Description |
|---|---|---|
| Momelotinib treatment | EXPERIMENTAL | Patients will receive momelotinib. |
| Name | Type | Description |
|---|---|---|
| Momelotinib treatment | DRUG | Patients included will receive momelotinib continuously until disease progression or loss of response, at physician's discretion. |
Inclusion Criteria: * ECOG (Eastern Cooperative Oncology Group) performance status 0-2 at the time of screening * Age ≥ 18 years * Written informed consent * Diagnosis of VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome with UBA1 (Ubiquitin Like Modifier Activating Enzym...
Momelotinib is an investigational small molecule being studied for VEXAS Syndrome, Myelodysplastic Syndromes, and Primary Myelofibrosis (PMF). It is also evaluated in related myeloproliferative conditions such as post-polycythemia vera and post-essential thrombocythemia myelofibrosis. It is not approved and remains in clinical development.
Momelotinib is a kinase inhibitor, belonging to the -tinib class of drugs that block specific enzymes involved in cell signaling. Its development focuses on myelofibrosis and other hematologic conditions, where kinase activity drives disease progression.
Momelotinib is developed by GSK plc, a global biopharma company traded on the New York Stock Exchange under the ticker GSK. GSK is conducting clinical trials to evaluate the drug's safety and efficacy in myelofibrosis and related blood disorders.
Momelotinib is in Phase 2 clinical development for VEXAS Syndrome and Myelodysplastic Syndromes. It has completed Phase 1, Phase 2, and Phase 3 trials in Primary Myelofibrosis, including a randomized Phase 3 study comparing it to ruxolitinib.
Momelotinib has been studied in six clinical trials with 963 total participants. Notable trials include NCT01236638, a completed Phase 2 extension study in Primary Myelofibrosis, and NCT01969838, a completed Phase 3 trial comparing momelotinib to ruxolitinib in myelofibrosis.
Yes, momelotinib treatment refers to the same drug, momelotinib. The term is used interchangeably in clinical contexts to describe the therapy being evaluated for conditions like Primary Myelofibrosis and VEXAS Syndrome.