Clinical trial NCT05061147: 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)
Official title: A Single-arm, Multi-Center, Phase Ib/Ⅱ Clinical Trial of Max-40279-01 in Combination With Azacitidine (AZA) in Adult Patients With Myelodysplastic Syndrome (MDS) or Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Leukemia
Acute Myeloid Leukemia
Relapsed/Refractory Acute Myeloid Leukemia
Myelodysplastic Syndrome
Keywords: Relapsed/Refractory Acute Myeloid Leukemia
Myelodysplastic Syndrome
Lead sponsor: Maxinovel Pty., Ltd.
Sponsor class: INDUSTRY
First posted: 2021-09-29
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

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

Eligibility criteria: Inclusion Criteria:

1. Males and/or females over age 18
2. 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);
3. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
4. Expected survival \>3 months.
5. 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.
6. Signed informed consent form.

Exclusion Criteria:

1. Acute promyelocytic leukemia according to World Health Organization 2016 criteria
2. Known central nervous system involvement
3. Medical history of difficulty swallowing, malabsorption or other chronic gastrointestinal disease, or conditions that may hamper compliance and/or absorption of the tested product
4. Known allergies, hypersensitivity, or intolerance to Max-40279-01 or AZA or the excipients of these treatments
5. 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

Primary outcomes:
measure: Maximum tolerated dose (MTD)
timeFrame: Through study Part 1 completion, an average of 6 months
measure: Phase II dose (RP2D)
timeFrame: Through study Part 1 completion, an average of 6 months
measure: Overall survival(OS)
timeFrame: Up to 24 months
measure: Rate of complete remission (CRc)
timeFrame: Up to 24 months
Secondary outcomes:
measure: Tmax
timeFrame: Approximately 4 weeks
measure: Cmax
timeFrame: Approximately 4 weeks
measure: AUC
timeFrame: Approximately 4 weeks
measure: t1/2
timeFrame: Approximately 4 weeks
measure: Objective response rate (ORR)
timeFrame: 1 months (anticipated)
measure: Safety and tolerability assessed by incidence and severity of adverse events
timeFrame: 24 months
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital
Study officials:
References: