Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
KPT-330 · 2 trials · 10 indications
Toxicities will be tabulated by type and grade.
Toxicities graded by CTCAE version 4.0
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product during the course of a study and which does not necessarily have to have a causal relationship with this treatment. An Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. TEAEs are any untoward medical incidence in a participant during administered study treatment, whether or not these events were related to study treatment.
Recommended Phase 2 dose was determined by maximum tolerated dose (MTD). MTD was defined as the next lower dose level below the one in which \> 1 of 3 participants or ≥ 2 of 6 participants experienced dose-limiting toxicity (DLT), provided that that dose level is ≤25% lower than the highest dose tested. If the projected MTD was \>25% lower than the highest dose tested, then an additional cohort of ≥3 participants were added at a dose that was intermediate between the intolerable dose and the next lower dose.
| Arm | Type | Description |
|---|---|---|
| KPT-330 | EXPERIMENTAL | KPT-330 will be administered twice a week on Days 1 and 3 for four weeks. Starting dose 30 mg/m2.In the dose-escalation cohort, three patients will initially be enrolled at each dose level and will be monitored for a DLT during the 28-day treatment cycle before dose escalation may occur. |
| selinexor | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| KPT-330 | DRUG | - |
Inclusion Criteria: * Age: Patient must be ≥ 12 months (365 days) and ≤ 21 years. * Histologically confirmed diagnosis of relapsed or refractory ALL (including Burkitt leukemia), AML, mixed lineage leukemia, biphenotypic leukemia, or chronic myelogenous leukemia (CML) in blast crisis. * Refracto...
KPT-330 is an investigational small molecule being studied for the treatment of hematological malignancies, including relapsed acute lymphoblastic leukemia (ALL). It is currently in Phase 1 clinical development and is not yet approved by the FDA.
KPT-330 targets XPO1, a nuclear export protein. By inhibiting XPO1, the drug is designed to block the export of tumor suppressor proteins from the nucleus, potentially restoring their function in cancer cells.
KPT-330 is being developed by Karyopharm Therapeutics Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol KPTI.
KPT-330 is in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. Clinical trials are ongoing to evaluate its safety and efficacy in patients with hematological cancers.
KPT-330 has been studied in two Phase 1 trials. NCT01607892, a completed safety study in 286 adults with advanced hematological cancer, and NCT02091245, an active trial in 16 children with relapsed or refractory leukemias including ALL and AML.
KPT-330 is also known as selinexor. It is a selective inhibitor of nuclear export (SINE) compound being developed by Karyopharm Therapeutics for the treatment of hematological malignancies.