A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Max-40279-01 in Combination With Azacitidine (AZA) in Patients With Myelodysplastic Syndrome (MDS) or Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)

NCT05061147 | Unknown Phase 1

• 1 other identifier: MAX-40279-004
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a phase Ib/II study of Max-40279-01 in combination with Azacitidine (AZA) in patients with Myelodysplastic Syndrome (MDS) or Relapsed/Refractory Acute Myeloid Leukemia (R/R AML). This study include Phase Ib and Phase II study. The phase Ib study is designed to evaluate the safety and tolerability of MAX-40279-01 in combination with Azacitidine (AZA) in patients with Relapsed or Refractory AML. The phase II study is designed to preliminarily assess the efficacy and safety of Max-40279-01 in combination with Azacitidine (AZA) in patients with Myelodysplastic Syndrome (MDS) or Relapsed/Refractory Acute Myeloid Leukemia (R/R AML).

Conditions

Leukemia; Acute Myeloid Leukemia; Relapsed/Refractory Acute Myeloid Leukemia; Myelodysplastic Syndrome

Keywords

Relapsed/Refractory Acute Myeloid Leukemia; Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (4)

• Maximum tolerated dose (MTD)

  To explore the maximum tolerable dose (MTD) of Max-40279-01 in combination with Azacitidine (AZA) for patients with r/r AML or MDS, the recommended phase II dose (RP2D).

  Through study Part 1 completion, an average of 6 months

• Phase II dose (RP2D)

  Recommended phase II dose (RP2D)

  Through study Part 1 completion, an average of 6 months

• Overall survival(OS)

  Up to 24 months

• Rate of complete remission (CRc)

  including Complete Remission with incomplete Platelet recovery (CRp) and Complete Remission with incomplete hematologic recovery (CRi)

  Up to 24 months

Secondary Outcomes (6)

• Tmax

  Approximately 4 weeks

• Cmax

  Approximately 4 weeks

• AUC

  Approximately 4 weeks

• t1/2

  Approximately 4 weeks

• Objective response rate (ORR)

  1 months (anticipated)

Study Arms (1)

Max-40279-01 in combination with Azacitidine (AZA)

EXPERIMENTAL

This is an open-label Phase Ib/II clinical study. The study will be conducted in two parts: Part I: Phase Ib dose escalation. Participants receive Max-40279-01 in combination with azacytidine (AZA), with different dose schedules. Part II: Phase II dose expansion. Participants divide into positive group and negative group according to whether FLT3 gene mutation occurs, approximately 40 people per group. All participants receive the recommended dose for Part 2 of Max-40279-01 with azacytidine (AZA).

Drug: MAX-40279-01

Interventions

MAX-40279-01 DRUG

Drug: AZA AZA will be administered at 75 mg/m\^2 by subcutaneous injection for 7 consecutive days from D1 to D7 in 28-day treatment cycles. Other Name: Azacitidine Drug: Max-40279-01 Max-40279-01 will be administered as a combination of multiple oral capsules containing 5 and 25 mg. An alternate combination of 35 mg, 50 mg and 60 mg Max-40279-01 twice a day may be utilized. Other Name: NA

Also known as: Azacitidine (AZA)

Max-40279-01 in combination with Azacitidine (AZA)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and/or females over age 18
• A diagnosis of AML according to the World Health Organization (WHO) 2016 criteria with relapsed or refractory disease and have exhausted, or are ineligible for therapeutic options, or int-risk or high-risk or very high-risk MDS according to revised International Prognostic Scoring System (IPSS-R);
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
• Expected survival \>3 months.
• No radiotherapy, surgery or hormonal therapy for any kind of within 2 weeks prior to participating in this study. Patients must have fully recovered from the acute toxicities of any prior treatment with any anti-cancer drugs (including hypomethylating agents in MDS patients), radiotherapy or other anti-cancer modalities (i.e., returned to baseline status as noted before most recent treatment) for any tumors. Patients with persisting, stable chronic toxicities from such prior treatment ≤Grade 1 are eligible, but must be documented as such.
• Signed informed consent form.

You may not qualify if:

• Acute promyelocytic leukemia according to World Health Organization 2016 criteria
• Known central nervous system involvement
• Medical history of difficulty swallowing, malabsorption or other chronic gastrointestinal disease, or conditions that may hamper compliance and/or absorption of the tested product
• Known allergies, hypersensitivity, or intolerance to Max-40279-01 or AZA or the excipients of these treatments
• 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

Sponsors & Collaborators

• Maxinovel Pty., Ltd.: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, China

RECRUITING

MeSH Terms

Conditions

Leukemia; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Central Study Contacts

Hanying Bao, MD,Ph.D

CONTACT

+86-021-51370693; hybao@maxinovel.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2021
• First Posted: September 29, 2021
• Study Start: September 16, 2021
• Primary Completion: April 30, 2022
• Study Completion: October 31, 2022
• Last Updated: October 1, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)