Study of MAX-40279 in Patients With Relapsed or Refractory Acute Myelogenous Leukemia (AML)

A Phase I Study for Tolerance and Pharmacokinetic Characteristic of MAX-40279 in Patients With Relapsed or Refractory Acute Myelogenous Leukemia

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.

Study Overview

• Status: Recruiting
• Conditions: AML
• Intervention / Treatment: Drug: MAX-40279-01

Detailed Description

This is a two-part study comprised of a dose escalation part and a dose expansion part.

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310003
      • Recruiting
      • The First Affiliated Hospital, Zhejiang University
      • Contact: Zhen Cai, MD,PhD; Email: caiz@zju.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: MAX-40279-01	Drug: MAX-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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events (AEs)	Incidence of treatment-related AEs	8 weeks
Maximum tolerated dose (MTD）	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.	4 weeks
Phase II dose (RP2D)	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.	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tmax	Time to maximum plasma concentration	Approximately 4 weeks
AUC	Area under the time-concentration curve	Approximately 4 weeks
t1/2	Observed terminal half-life	Approximately 4 weeks
Cmax	Maximum plasma drug concentration	Approximately 4 weeks
Objective response rate (ORR)	The ORR is defined as the proportion of subjects with confirmed CR or confirmed PR, based on IRWG.	1 months (anticipated)

Collaborators and Investigators

• Sponsor: Maxinovel Pty., Ltd.

Study record dates

Study Major Dates

• Study Start (ACTUAL): April 16, 2019
• Primary Completion (ANTICIPATED): December 1, 2022
• Study Completion (ANTICIPATED): December 1, 2022

Study Registration Dates

• First Submitted: December 3, 2019
• First Submitted That Met QC Criteria: December 3, 2019
• First Posted (ACTUAL): December 5, 2019

Study Record Updates

• Last Update Posted (ACTUAL): January 19, 2022
• Last Update Submitted That Met QC Criteria: January 18, 2022
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: MAX-40279-002

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No