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Leukemia, Myeloid, AcutePhase 1NCT04241549

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

A Phase 1 study testing Cusatuzumab and Azacitidine in people with Leukemia, Myeloid, Acute, run by OncoVerity, Inc. at 5 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 20 years and older
Conditions the study is for
Leukemia, Myeloid, Acute
What is being tested?
Cusatuzumab and Azacitidine
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
5 sitesJapan
Study dates
25 Mar 2020to 19 Jul 2021
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 20 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

Japan5 sites

  • Fukushima Medical University Hospital

    Fukushima, 960-1295

  • Gunmaken Saiseikai Maebashi Hospital

    Maebashi, 371-0821

  • Osaka City General Hospital

    Osaka, 534-0021

  • NTT Medical Center Tokyo

    Tokyo, 141-8625

  • University of Fukui Hospital

    Yoshida, 910-1193

Other trials in Japan

About this study

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

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.

Being tested
Cusatuzumab, Azacitidine
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CusatuzumabDrug

    Also called JNJ-74494550, ARGX-110

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

  • AzacitidineDrug

    Also called VIDAZA

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

Study groups in NCT04241549
Study groupRoleWhat they receive
Part 1 (Dose Finding): Cusatuzumab + AzacitidineExperimentalParticipants 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.
Receives: Drug: Cusatuzumab; Drug: Azacitidine
Part 2 (Dose Expansion): Cusatuzumab + AzacitidineExperimentalParticipants 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.
Receives: Drug: Cusatuzumab; Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Up to 3 years

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

    Number of participants with DLTs will be reported.

    Measured: Up to 42 days

  3. 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.

    Measured: Up to 42 days

Secondary outcomes (10)

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

    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.

    Measured: Up to 9 months

  2. Part 1: Objective Response Rate (ORR)

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

    Measured: Up to 6 months

  3. Part 2: Objective Response Rate (ORR)

    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.

    Measured: Up to 9 months

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

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

    Measured: Up to 9 months

  5. Part 1 and Part 2: Time to Response

    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..

    Measured: Up to 3 years

  6. Part 1 and Part 2: Duration of Response

    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.

    Measured: Up to 3 years

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

    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.

    Measured: Up to 3 years

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

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

    Measured: Up to 3 years

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

    Cmax is the maximum observed serum concentration.

    Measured: Up to 3 years

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

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

    Measured: Up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Jan 2020
  2. Study start25 Mar 2020
  3. Primary completion19 Jul 2021
  4. Study completion19 Jul 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04241549
Sponsor's protocol ID
CR108732
Other IDs
74494550AML1002
Status last verified
Aug 2023
First submitted
23 Jan 2020
Last updated
9 Aug 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Cusatuzumab and Azacitidine in people with Leukemia, Myeloid, Acute, run by OncoVerity, Inc. at 5 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part 1 and Part 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Up to 3 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04241549.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Aug 2023.

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