AML
Conditions
Brief summary
This is a non-randomized, open-label, single-arm, dose-escalation Phase I study to evaluate the safety and tolerability of MAX-40279-01 in patients with Relapsed or Refractory AML.
Detailed description
This is a two-part study comprised of a dose escalation part and a dose expansion part.
Interventions
DRUGMAX-40279-01
Part 1: Dose escalation, MAX-40279-01 twice daily with dose modifications based on tolerability criteria. Part 2: Dose expansion, Recommended doses from Part 1. For each dose level, a single dose of MAX-40279-01 will be first administered orally followed by 1 day observation, then continuous treatment will start 4 weeks treatment (per cycle).
Sponsors
Maxinovel Pty., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Males and/or females over age 18. * Subject has morphologically documented relapsed or refractoryprimary AML as defined by the World Health Organization (WHO) 2016 criteria for which no established standard therapy is available. * ECOG performance status of 0 to 2. * Persistent chronic clinically significant non-hematological toxicities from prior treatment (including chemotherapy, kinase inhibitors, immunotherapy, experimental agents, radiation, HSCT, or surgery) must be Grade ≤ 1. * Any anti-tumor treatment with radiation therapy, surgery, or immunotherapy wihtin 2 weeks prior to trial entry. * Life expectancy of at least 3 months. * Both men and women of reproductive potential must agree to use a highly effective method of birth control during the study and for six months following the last dose of study drug.
Exclusion criteria
* Disease diagnosis of acute promyelocytic leukemia. * Medical history of difficulty swallowing, malabsorption or other chronic gastrointestinal disease, or conditions that may hamper compliance and/or absorption of the tested product. * Previously treated malignancies other than the current disease, except for adequately treated non-melanoma skin cancer, in situ cancer, or other cancer from which the subject has been disease-free for at least 5 years at the trial entry. * Laboratory values not within the Protocol-defined range. * Concomitant disease or condition that could interfere with the conduct of the trial, or that would, in the opinion of the Investigator, pose an unacceptable risk to the subject in this trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events (AEs) | 8 weeks | Incidence of treatment-related AEs |
| Maximum tolerated dose (MTD） | 4 weeks | MTD will be defined as the maximum dose level at which no more than 1 of 3 participants experience a dose-limiting toxicity (DLT) within the first 4 weeks of multiple dosing. |
| Phase II dose (RP2D) | 4 weeks | The number and proportion of patients experiencing at least 1 dose-limiting toxicity (DLT) will be used as the primary measure to evaluate the RP2D of MAX-40279-01. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| t1/2 | Approximately 4 weeks | Observed terminal half-life |
| Tmax | Approximately 4 weeks | Time to maximum plasma concentration |
| Objective response rate (ORR) | 1 months (anticipated) | The ORR is defined as the proportion of subjects with confirmed CR or confirmed PR, based on IRWG. |
| Cmax | Approximately 4 weeks | Maximum plasma drug concentration |
| AUC | Approximately 4 weeks | Area under the time-concentration curve |
Countries
China
Contacts
Primary ContactHanying Bao, MD,Ph.D
Outcome results
None listed