---
title: 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
nct_id: NCT04241549
overall_status: COMPLETED
phase: PHASE1
sponsor: OncoVerity, Inc.
study_type: INTERVENTIONAL
primary_condition: Leukemia, Myeloid, Acute
countries: Japan
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04241549.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04241549"
ct_last_update_post_date: 2023-08-09
last_seen_at: "2026-05-12T07:30:43.885Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# 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

**NCT ID:** [NCT04241549](https://clinicaltrials.gov/study/NCT04241549)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE1
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 6
- **Lead Sponsor:** OncoVerity, Inc.
- **Collaborators:** argenx, Janssen Pharmaceutical K.K.
- **Conditions:** Leukemia, Myeloid, Acute
- **Start Date:** 2020-03-25
- **Completion Date:** 2021-07-19
- **CT.gov Last Update:** 2023-08-09

## 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

- **Minimum age:** 20 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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
```

## Arms

- **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.
- **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.

## Interventions

- **Cusatuzumab** (DRUG) — Cusatuzumab at a dose 20 milligram per kilogram (mg/kg) once every 2 weeks will be administered intravenously.
- **Azacitidine** (DRUG) — Azacitidine at a dose 75 milligram per square meters (mg/m\^2) will be administered subcutaneously or intravenously.

## Primary Outcomes

- **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.
- **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.
- **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

- **Part 1 and Part 2: Percentage of Participants with Complete Response (CR)** _(time frame: Up to 9 months)_
- **Part 1: Objective Response Rate (ORR)** _(time frame: Up to 6 months)_
- **Part 2: Objective Response Rate (ORR)** _(time frame: Up to 9 months)_
- **Part 2: Percentage of Participants with Hematologic Improvement (HI)** _(time frame: Up to 9 months)_
- **Part 1 and Part 2: Time to Response** _(time frame: Up to 3 years)_
- **Part 1 and Part 2: Duration of Response** _(time frame: Up to 3 years)_
- **Part 1 and Part 2: Red Blood Cell (RBC) or Platelets Transfusion Independence** _(time frame: Up to 3 years)_
- **Part 1 and Part 2: Overall Survival (OS)** _(time frame: Up to 3 years)_
- **Part 1 and Part 2: Maximum Serum Concentration (Cmax) of Cusatuzumab** _(time frame: Up to 3 years)_
- **Part 1 and Part 2: Serum Trough Concentration (Ctrough) of Cusatuzumab** _(time frame: Up to 3 years)_

## Locations (5)

- Fukushima Medical University Hospital, Fukushima, Japan
- Gunmaken Saiseikai Maebashi Hospital, Maebashi, Japan
- Osaka City General Hospital, Osaka, Japan
- NTT Medical Center Tokyo, Tokyo, Japan
- University of Fukui Hospital, Yoshida, Japan

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.fukushima medical university hospital|fukushima||japan` — added _(2026-05-12)_
- `locations.gunmaken saiseikai maebashi hospital|maebashi||japan` — added _(2026-05-12)_
- `locations.osaka city general hospital|osaka||japan` — added _(2026-05-12)_
- `locations.ntt medical center tokyo|tokyo||japan` — added _(2026-05-12)_
- `locations.university of fukui hospital|yoshida||japan` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT04241549.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT04241549*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*