Skip to main content
OpenTrials
Completed

NCT Number: 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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Leukemia, Myeloid, Acute Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Neoplasms Neoplasms by Histologic Type

Age range

20 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Fukushima Medical University Hospital, Fukushima, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Cusatuzumab

Drug

Cusatuzumab at a dose 20 milligram per kilogram (mg/kg) once every 2 weeks will be administered intravenously.

Other names: JNJ-74494550, ARGX-110

Azacitidine

Drug

Azacitidine at a dose 75 milligram per square meters (mg/m^2) will be administered subcutaneously or intravenously.

Other names: VIDAZA

Primary outcomes

  1. Part 1 and Part 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Up to 3 years

    Number of participants with AEs and SAEs will be reported.

  2. Part 1 and Part 2: Number of Participants with Dose-Limiting Toxicity (DLTs)

    Time frame: Up to 42 days

    Number of participants with DLTs will be reported.

  3. Part 1 and Part 2: Severity of DLT as Assessed by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE)

    Time frame: Up to 42 days

    Severity of DLT as assessed by NCI-CTCAE in participants will be reported.

Secondary outcomes

  1. Part 1 and Part 2: Percentage of Participants with Complete Response (CR)

    Time frame: Up to 9 months

    Percentage of participants with complete response based on response criteria per investigator assessment in participants with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) will be reported.

  2. Part 1: Objective Response Rate (ORR)

    Time frame: Up to 6 months

    ORR is defined as percentage of participants with CR, CRh and CRi based on response criteria per investigator assessment in participants with AML.

  3. Part 2: Objective Response Rate (ORR)

    Time frame: Up to 9 months

    ORR is defined as the percentage of participants with CR, partial response (PR) and marrow CR based on response criteria per investigator assessment in participants with MDS.

  4. Part 2: Percentage of Participants with Hematologic Improvement (HI)

    Time frame: Up to 9 months

    Percentage of participants with hematologic improvement will be reported according to response criteria per investigator assessment in participants with MDS.

  5. Part 1 and Part 2: Time to Response

    Time frame: Up to 3 years

    Time to response is defined as time from first dose to achieving the first response of CR, CRh, or CRi in participants with AML and CR, PR or marrow CR in participants with MDS..

  6. Part 1 and Part 2: Duration of Response

    Time frame: Up to 3 years

    Duration of response is defined as time from achieving the first response of CR, CRh, or CRi in participants with AML and CR, PR or marrow CR in participants with MDS to hematologic relapse or death of any cause.

  7. Part 1 and Part 2: Red Blood Cell (RBC) or Platelets Transfusion Independence

    Time frame: Up to 3 years

    Transfusion independence (RBC or platelets) is defined as a period of at least 56 consecutive days with no transfusion between first dose of study drug and the last dose of study drug +30 days.

  8. Part 1 and Part 2: Overall Survival (OS)

    Time frame: Up to 3 years

    OS is defined as the time from initial study intervention administration to death from any cause.

  9. Part 1 and Part 2: Maximum Serum Concentration (Cmax) of Cusatuzumab

    Time frame: Up to 3 years

    Cmax is the maximum observed serum concentration.

  10. Part 1 and Part 2: Serum Trough Concentration (Ctrough) of Cusatuzumab

    Time frame: Up to 3 years

    Ctrough is the serum concentration immediately prior to the next drug administration.

Sponsors and collaborators

Lead sponsor

OncoVerity, Inc.

Industry

Collaborators

  • Janssen Pharmaceutical K.K.
  • argenx

Registry information

Official study 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

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Jan 27, 2020
Registry last updated
Aug 9, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.