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

NCT04241549 | Completed Phase 1 FDA Drug

• 2 other identifiers: CR10873274494550AML1002
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 5

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.

Conditions

Leukemia, Myeloid, Acute

Outcome Measures

Primary Outcomes (3)

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

  Number of participants with AEs and SAEs will be reported.

  Up to 3 years

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

  Number of participants with DLTs will be reported.

  Up to 42 days

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

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

  Up to 42 days

Secondary Outcomes (10)

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

  Up to 9 months

• Part 1: Objective Response Rate (ORR)

  Up to 6 months

• Part 2: Objective Response Rate (ORR)

  Up to 9 months

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

  Up to 9 months

• Part 1 and Part 2: Time to Response

  Up to 3 years

Study Arms (2)

Part 1 (Dose Finding): Cusatuzumab + Azacitidine

EXPERIMENTAL

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; Drug: Azacitidine

Part 2 (Dose Expansion): Cusatuzumab + Azacitidine

EXPERIMENTAL

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; Drug: Azacitidine

Interventions

Cusatuzumab DRUG

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

Also known as: JNJ-74494550, ARGX-110

Part 1 (Dose Finding): Cusatuzumab + Azacitidine; Part 2 (Dose Expansion): Cusatuzumab + Azacitidine

Azacitidine DRUG

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

Also known as: VIDAZA

Part 1 (Dose Finding): Cusatuzumab + Azacitidine; Part 2 (Dose Expansion): Cusatuzumab + Azacitidine

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• OncoVerity, Inc.: lead
• argenx: collaborator
• Janssen Pharmaceutical K.K.: collaborator

Study Sites (5)

Fukushima Medical University Hospital

Fukushima, 960-1295, Japan

Location

Gunmaken Saiseikai Maebashi Hospital

Maebashi, 371-0821, Japan

Location

Osaka City General Hospital

Osaka, 534-0021, Japan

Location

NTT Medical Center Tokyo

Tokyo, 141-8625, Japan

Location

University of Fukui Hospital

Yoshida, 910-1193, Japan

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

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

Study Officials

• Janssen Pharmaceutical K.K., Japan Clinical Trial

  Janssen Pharmaceutical K.K.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2020
• First Posted: January 27, 2020
• Study Start: March 25, 2020
• Primary Completion: July 19, 2021
• Study Completion: July 19, 2021
• Last Updated: August 9, 2023

Record last verified: 2023-08

Locations

JP (5)