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KPT-9274 · 1 trial · 3 indications
Dose escalation will continue until the MTD is determined. The MTD is defined as the highest dose at which ≤1 patient experiences a DLT in Cycle 1. Count of participants who did not experience a DLT are listed in outcome measure data table.
| Arm | Type | Description |
|---|---|---|
| De-escalation Cohort; 20mg | ACTIVE_COMPARATOR | For the purposes of dose escalation decisions, a standard 3+3 dose escalation design will be used.The initial cohort, Cohort 1, will consist of 3 enrolled patients who will be treated at 30 mg. If the MTD is exceeded at cohort 1, de-escalation to cohort 0 (20 mg) will occur. If the MTD is not exceeded in cohort 1, dose escalation will continue based on a standard 3+3 design at the dose levels. |
| Cohort 1; 30 mg | ACTIVE_COMPARATOR | The initial cohort, Cohort 1, will consist of 3 enrolled patients who will be treated at 30 mg. |
| Cohort 2; 40mg | ACTIVE_COMPARATOR | Dose escalation to 40 mg after initial cohort (Cohort 1) after a minimum of 1 cycle of treatment, defined as receiving ≥75% of KPT-9274 doses during Cycle 1 (e.g., ≥9 of 12 doses in the 3 doses/week schedule), or have a DLT within the first cycle of treatment to be evaluable for dose escalation decisions. |
| Cohort 3; 60mg | ACTIVE_COMPARATOR | Dose escalation to 60 mg after initial cohort (Cohort 1) after a minimum of 1 cycle of treatment, defined as receiving ≥75% of KPT-9274 doses during Cycle 1 (e.g., ≥9 of 12 doses in the 3 doses/week schedule), or have a DLT within the first cycle of treatment to be evaluable for dose escalation decisions. |
| Cohort 4; 80mg | ACTIVE_COMPARATOR | Dose escalation to 80 mg after initial cohort (Cohort 1) after a minimum of 1 cycle of treatment, defined as receiving ≥75% of KPT-9274 doses during Cycle 1 (e.g., ≥9 of 12 doses in the 3 doses/week schedule), or have a DLT within the first cycle of treatment to be evaluable for dose escalation decisions. |
| Cohort 5; 100mg | ACTIVE_COMPARATOR | Dose escalation to 100 mg after initial cohort (Cohort 1) after a minimum of 1 cycle of treatment, defined as receiving ≥75% of KPT-9274 doses during Cycle 1 (e.g., ≥9 of 12 doses in the 3 doses/week schedule), or have a DLT within the first cycle of treatment to be evaluable for dose escalation decisions. |
| Name | Type | Description |
|---|---|---|
| KPT-9274 | DRUG | KPT-9274 is a first-in-class orally bioavailable, non-competitive, small molecule, dual modulator of p21 protein (Cdc42/Rac)-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase /PBEF/visfatin (NAMPT). NAMPT is the rate-limiting enzyme in the metabolic scavenging pathway that utilizes nicotinamide to replenish nicotinamide adenine dinucleotide (NAD), an essential metabolic cofactor and second messenger. Recent investigations have shown that in in vitro and in vivo models, NAMPT is uniquely essential for relapsed or refractory AML stem cells. Targeting relapsed AML stem cells, through targeting of NAMPT, may allow for a promising therapeutic opportunity for patients with relapsed or refractory AML. |
Inclusion Criteria: 1. Written informed consent obtained prior to any study related procedures required solely for this research study. 2. Age ≥18 years. 3. Patients with WHO-confirmed non-APL AML who have not responded to or relapsed after at least one prior therapy and for whom no standard therap...
KPT-9274 is an investigational small molecule being studied for the treatment of acute myeloid leukemia (AML), specifically in patients with relapsed and refractory disease. It is being developed by Karyopharm Therapeutics Inc. (NASDAQ: KPTI) and is currently in Phase 1 clinical development.
KPT-9274 is being developed by Karyopharm Therapeutics Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol KPTI. The company is conducting clinical research on this investigational small molecule for the treatment of acute myeloid leukemia.
KPT-9274 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. The drug is being studied in patients with relapsed and refractory acute myeloid leukemia, and its Phase 1 trial has been completed.
KPT-9274 has one completed Phase 1 clinical trial, identified as NCT04914845. This trial, titled "KPT-9274 in Patients With Relapsed and Refractory Acute Myeloid Leukemia," enrolled 16 participants in the United States. The study was active-controlled and included biomarker-selected patients.
KPT-9274 is a distinct investigational small molecule developed by Karyopharm Therapeutics Inc. It is not known to be marketed under any other name. The drug is being studied specifically for acute myeloid leukemia and is not approved for any use.