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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

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ClinicalTrials.gov Identifier: NCT04241549
Recruitment Status : Recruiting
First Posted : January 27, 2020
Last Update Posted : May 29, 2020
Sponsor:
Collaborator:
argenx BVBA
Information provided by (Responsible Party):
Janssen Pharmaceutical K.K.

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.

Condition or disease Intervention/treatment Phase
Leukemia, Myeloid, Acute Drug: Cusatuzumab Drug: Azacitidine Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: 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
Actual Study Start Date : March 25, 2020
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : March 31, 2023


Arm Intervention/treatment
Experimental: Part 1 (Dose Finding): Cusatuzumab + Azacitidine
Participants with acute myeloid leukemia (AML) will receive cusatuzumab intravenously (IV) in combination with azacitidine subcutaneously (SC) or IV. The dose levels will be escalated based on the decisions of the Study Evaluation Team (SET) until the recommended Phase 2 Dose (RP2D) has been identified.
Drug: Cusatuzumab
Cusatuzumab at a dose 20 milligram per kilogram (mg/kg) once every 2 weeks will be administered intravenously.
Other Names:
  • JNJ-74494550
  • ARGX-110

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

Experimental: Part 2 (Dose Expansion): Cusatuzumab + Azacitidine
Participants with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) will receive cusatuzumab intravenously (IV) at the recommended Phase 2 dose (RP2D) determined in Part 1 in combination with azacitidine subcutaneously (SC) or IV.
Drug: Cusatuzumab
Cusatuzumab at a dose 20 milligram per kilogram (mg/kg) once every 2 weeks will be administered intravenously.
Other Names:
  • JNJ-74494550
  • ARGX-110

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




Primary Outcome Measures :
  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 Outcome Measures :
  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.



Information from the National Library of Medicine

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Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 a 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 azacitidine
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04241549


Contacts
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Contact: Study Contact 844-434-4210 JNJ.CT@sylogent.com

Locations
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Japan
Fukushima Medical University Hospital Recruiting
Fukushima, Japan, 960-1295
Gunmaken Saiseikai Maebashi Hospital Recruiting
Maebashi, Japan, 371-0821
Osaka City General Hospital Recruiting
Osaka, Japan, 534-0021
Hokkaido University Hospital Not yet recruiting
Sapporo, Japan, 060-8648
NTT Medical Center Tokyo Recruiting
Tokyo, Japan, 141-8625
University of Fukui Hospital Recruiting
Yoshida, Japan, 910-1193
Sponsors and Collaborators
Janssen Pharmaceutical K.K.
argenx BVBA
Investigators
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Study Director: Janssen Pharmaceutical K.K., Japan Clinical Trial Janssen Pharmaceutical K.K.
Additional Information:
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Responsible Party: Janssen Pharmaceutical K.K.
ClinicalTrials.gov Identifier: NCT04241549    
Other Study ID Numbers: CR108732
74494550AML1002 ( Other Identifier: Janssen Pharmaceutical K.K., Japan )
First Posted: January 27, 2020    Key Record Dates
Last Update Posted: May 29, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency.

As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

URL: https://www.janssen.com/clinical-trials/transparency

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Preleukemia
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Azacitidine
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors