Clinical trial NCT04241549: A Study of Cusatuzumab Plus Azacitidine in Japanese Participants With Newly Diagnosed Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome Who Are Not Candidates for Intensive Treatment
Official title: A Phase 1 Study of Cusatuzumab Plus Azacitidine in Japanese Patients With Newly Diagnosed Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome Who Are Not Candidates for Intensive Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Leukemia, Myeloid, Acute
Lead sponsor: OncoVerity, Inc.
Sponsor class: INDUSTRY
Collaborators: name: argenx
name: Janssen Pharmaceutical K.K.
First posted: 2020-01-27
Enrollment: 6
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the recommended Phase 2 dose and evaluate safety profile of cusatuzumab in combination with azacitidine in Japanese participants with treatment naïve acute myeloid leukemia (AML) who are not candidates for intensive treatment.


Eligibility criteria: Inclusion Criteria:

* For acute myeloid leukemia (AML) participants: AML according to World Health Organization (WHO) 2016 criteria and fulfilling all of the following criteria:(a) more than or equal to (\>=) 75 years of age, or younger participants who are not eligible for or not willing to receive an intensive treatment (including stem cell transplantation) with curative intent and (b) previously untreated AML (except: emergency leukapheresis, low dose of cytarabine and/or hydroxyurea during the screening phase to control hyperleukocytosis but must be discontinued at least one day prior to start of cusatuzumab \[Part 1\] or azacitidine \[Part 2\]). All trans retinoic acid (ATRA) treatment for presumed acute promyelocytic leukemia (APL) is permitted but must be discontinued at least 1 day prior to the start of cusatuzumab (Part 1) or azacitidine (Part 2)
* For Myelodysplastic Syndrome (MDS) participants (only for Part 2): MDS according to WHO 2016 criteria and fulfilling all of the following criteria: (a) Not eligible for or not willing to receive allogenic stem cell transplantation,(b) very high or high-risk MDS according to Revised International Prognostic Scoring System (IPSS-R) and (c) previously untreated MDS (except: transfusion and/or cytokine therapy including erythropoietin)
* Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1 or 2
* Must sign an informed consent form (ICF) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study
* A woman of childbearing potential must have a negative highly sensitive serum (beta human chorionic gonadotropin \[beta hCG\]) or urine pregnancy at screening

Exclusion Criteria:

* Acute promyelocytic leukemia (APL) with t (15;17), or its molecular equivalent promyelocytic leukemia retinoic acid receptor (PML RAR alpha)
* Leukemic involvement or clinical symptoms of leukemic involvement of the central nervous system
* Known allergies, hypersensitivity, or intolerance to cusatuzumab or azacitidine or its excipients (example, mannitol, an excipient of azacitidine)
* Prior treatment with a hypomethylating agent for treatment of AML or MDS
* A diagnosis of other malignancy that requires concurrent nonsurgical treatment

Primary outcomes:
measure: Part 1 and Part 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Up to 3 years
measure: Part 1 and Part 2: Number of Participants with Dose-Limiting Toxicity (DLTs)
timeFrame: Up to 42 days
measure: Part 1 and Part 2: Severity of DLT as Assessed by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE)
timeFrame: Up to 42 days
Secondary outcomes:
measure: Part 1 and Part 2: Percentage of Participants with Complete Response (CR)
timeFrame: Up to 9 months
measure: Part 1: Objective Response Rate (ORR)
timeFrame: Up to 6 months
measure: Part 2: Objective Response Rate (ORR)
timeFrame: Up to 9 months
measure: Part 2: Percentage of Participants with Hematologic Improvement (HI)
timeFrame: Up to 9 months
measure: Part 1 and Part 2: Time to Response
timeFrame: Up to 3 years
measure: Part 1 and Part 2: Duration of Response
timeFrame: Up to 3 years
measure: Part 1 and Part 2: Red Blood Cell (RBC) or Platelets Transfusion Independence
timeFrame: Up to 3 years
measure: Part 1 and Part 2: Overall Survival (OS)
timeFrame: Up to 3 years
measure: Part 1 and Part 2: Maximum Serum Concentration (Cmax) of Cusatuzumab
timeFrame: Up to 3 years
measure: Part 1 and Part 2: Serum Trough Concentration (Ctrough) of Cusatuzumab
timeFrame: Up to 3 years
Other outcomes:
Locations:
city: Fukushima
country: Japan
facility: Fukushima Medical University Hospital
city: Maebashi
country: Japan
facility: Gunmaken Saiseikai Maebashi Hospital
city: Osaka
country: Japan
facility: Osaka City General Hospital
city: Tokyo
country: Japan
facility: NTT Medical Center Tokyo
city: Yoshida
country: Japan
facility: University of Fukui Hospital
Study officials:
affiliation: Janssen Pharmaceutical K.K.
name: Janssen Pharmaceutical K.K., Japan Clinical Trial
role: STUDY_DIRECTOR
References: