A Study Comparing Venetoclax and Azacitidine Plus Cusatuzumab to Venetoclax and Azacitidine in Newly Diagnosed AML Ineligible for Intensive Therapy

NCT ID: NCT06384261

Last Updated: 2025-09-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-22

Study Completion Date

2027-06-30

Brief Summary

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The goal of this clinical trial is to learn if participants treated with the experimental drug cusatuzumab added to venetoclax and azacitidine works to treat acute myeloid leukemia (AML) compared to venetoclax and azacitidine. Venetoclax and azacitidine are drugs commonly used to treat AML in patients that are unable to receive chemotherapy to treat AML. The main question the clinical trial aims to answer is does cusatuzumab added to venetoclax and azacitidine prolong the length of time participants live compared to venetoclax and azacitidine?

Detailed Description

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This is a randomized, open-label, multicenter, Phase 2 trial to evaluate the efficacy, safety, and pharmacodynamics of cusatuzumab in combination with venetoclax and azacitidine compared to venetoclax and azacitidine in persons with newly diagnosed AML who are deemed ineligible for intensive chemotherapy.

Potential participants will be considered ineligible for intensive chemotherapy and, therefore, eligible for the study, if they meet the trial eligibility criteria and provide informed consent. Participants will undergo a diagnostic bone marrow biopsy and aspirate collected for pathology review, cytogenetics, fluorescence in situ hybridization (FISH), and polymerase chain reaction (PCR) analysis and other studies for confirmation of a diagnosis of AML and to define whether participants have adverse, intermediate, or favorable AML risk features. Participants will then be randomized 2:1 to either the experimental arm or the active comparator arm and stratified based on AML risk features (adverse, intermediate, and favorable risk).

Conditions

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Leukemia, Myeloid, Acute

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cusatuzumab in combination with venetoclax and azacitidine

Cusatuzumab 20 mg/kg administered intravenously on days 3 and 17 in combination with venetoclax administered orally up to 400 mg once daily and azacitidine 75 mg/m\^2 administered subcutaneously or intravenously once daily for 7 days of a 28 day cycle

Group Type EXPERIMENTAL

Cusatuzumab

Intervention Type DRUG

CD70 monoclonal antibody

Venetoclax

Intervention Type DRUG

BCL-2 inhibitor

Azacitidine

Intervention Type DRUG

Hypomethylating agent

Venetoclax in combination with azacitidine

Venetoclax administered orally up to 400 mg once daily and azacitidine 75 mg/m\^2 administered subcutaneously or intravenously once daily for 7 days of a 28 day cycle

Group Type ACTIVE_COMPARATOR

Venetoclax

Intervention Type DRUG

BCL-2 inhibitor

Azacitidine

Intervention Type DRUG

Hypomethylating agent

Interventions

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Cusatuzumab

CD70 monoclonal antibody

Intervention Type DRUG

Venetoclax

BCL-2 inhibitor

Intervention Type DRUG

Azacitidine

Hypomethylating agent

Intervention Type DRUG

Other Intervention Names

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OV-1001

Eligibility Criteria

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Inclusion Criteria

* Men and women ≥18 years old
* 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
* Diagnosis of AML according to ICC 2022 (with the exclusion of MDS/AML with 10-19% blasts)
* Previously untreated AML except may have received emergency leukapheresis, hydroxyurea before study entry to control hyperleukocytosis
* Deemed unfit for intensive chemotherapy by meeting at least 1 of the following criteria:

1. Participant is ≥75 years of age with Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 OR
2. Participant is ≥18 to 74 years of age and has any of the following comorbidities:

1. ECOG performance status of 2 or 3
2. Cardiac status including any one of the following: congestive heart failure requiring treatment or ejection fraction ≤50% or chronic stable angina
3. Known history of diffusion capacity of lung for carbon monoxide (DLCO) ≤65% of forced expiratory volume in the first second (FEV1) ≤65%
4. Creatinine clearance (CrCl) ≥15 mL/min to \<45 mL/min
5. Hepatic disorder with total bilirubin \>1.5 to 3x the upper limit of normal (ULN)
6. Any other comorbidity that the investigators determine to be incompatible with conventional intensive chemotherapy
* Adequate liver and renal function defined as:

1. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \<3xULN; for participants with leukemic infiltration of the liver (documented by biopsy or imaging), AST and ALT \<5xULN is permitted
2. Total bilirubin ≤1.5xULN, unless bilirubin rise is due to Gilbert's syndrome or of nonhepatic origin. Participants who are \<75 years of age may have a bilirubin up to 3xULN.
3. Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2 (by the Modification of Diet in Renal Disease \[MDRD\] formula). Participants who are \<75 years of age may have an eGFR ≥15 mL/min/1.73 m2.
* Women of childbearing potential (WOCBP), defined as fertile women between menarche and post menopause unless permanently sterile, must have a negative highly sensitive serum β-human chorionic gonadotropin (β-hCG) or urine pregnancy test at screening
* Must be willing to use contraception as consistent with institutional guidelines regarding the use of contraceptive methods for participants participating in clinical studies

1. WOCBP must agree to adhere to the following birth control measures while receiving study treatment continuing to 3 months after the last dose of study drug:

1. Must be practicing a highly effective method of birth control (failure rate of \<1% per year when used consistently and correctly) as determined by institutional standards
2. Must agree to not donate eggs (ova, oocytes) for the purposes of assisted reproduction
3. Must not be breastfeeding and not planning to become pregnant
2. Male participants who are sexually active with WOCBP, and male partners of study participants who are WOCBP, and who are not surgically or otherwise sterile must agree to adhere to the following birth control measures while receiving study treatment and for 3 months after the last dose of study drug:

1. Must agree to use a barrier method of birth control (e.g., either condom with spermicidal foam/gel/film/cream/suppository or partner with occlusive cap \[diaphragm or cervical/vault caps\] with spermicidal foam, gel, film, cream, or suppository)
2. Must not donate sperm
3. Must no plan to father a child
* Participants with HIV infection are eligible for the trial if the following criteria are met:

1. CD4+ T-cell count ≥200 cells/μL
2. No prior history of AIDS-defining opportunistic infection within the past 12 months
3. Receiving treatment with antiretroviral therapy
4. Undetectable viral load within 6 months of screening

Exclusion Criteria

* Any prior treatment for AML (except those outlined in inclusion criterion #4)
* Participant has received a hypomethylating agent (HMA) or venetoclax for MDS or myeloproliferative neoplasm
* Leukemic involvement in the central nervous system
* Participants with acute promyelocytic leukemia (APL)
* ECOG performance status of 4 for participants 18 to 74 years of age and ECOG performance status of 3 or 4 for participants ≥75 years of age
* Use of immune suppressive agents ≤4 weeks before the first administration of cusatuzumab. Participants may be included if free of systemic corticosteroids \>5 days before the first administration of cusatuzumab with the exception of corticosteroids at physiologic replacement doses.
* Received a live, attenuated vaccine within 4 weeks prior to initiation of study drug
* Active malignancies (i.e., progressing or requiring treatment change in the last 24 months) other than the disease being treated under study. Exceptions to this exclusion criterion include the following:

1. Nonmelanoma skin cancer treated within the last 24 months that is considered completely cured
2. Adequately treated breast lobular carcinoma in situ and breast ductal carcinoma in situ
3. Adequately treated cervical carcinoma in situ and breast ductal carcinoma in situ
4. History of localized breast cancer and receiving anti-hormonal agents, or history of localized prostate cancer (N0M0) and receiving androgen depravation therapy
5. A malignancy that is considered cured with minimal risk of recurrence
* Any active systemic infection
* History of prior HSCT (allogeneic or autologous transplants)
* Active hepatitis B or C infection or other clinically active liver diseases ad defined below:

1. Seropositivity for hepatitis B is defined by a positive test for hepatitis B surface antigen (HBsAg)
2. Participants with resolved infection (i.e., participants who are HBsAg negative with antibodies to total hepatitis B core antigen \[anti-HBc\] with or without the presence of hepatitis B surface antibody \[anti-HBs\]) must be screened using PCR measurement of hepatitis B virus (HBV) DNA levels. Those who are PCR positive will be excluded.

* Participants with serologic findings suggestive of HBV vaccination (anti-HBs positivity as the only serologic marker) AND a known history of prior HBV vaccination, do not need to be tested for HBV DNA by PCR
3. Active hepatitis C infection as defined by being positive for a nucleic acid test for hepatitis C virus (HCV) RNA
* Congestive hear failure severity that is New York Heart Association Class III or IV
* Unstable angina
* Known allergies, hypersensitivity, or intolerance to cusatuzumab, venetoclax, or azacitidine or their excipients (e.g., mannitol, an excipient of azacitidine)
* Inability or difficulty swallowing capsules/tablets, malabsorption syndrome, or any disease or medical condition significantly affecting gastrointestinal function
* Any condition for which, in the investigator's opinion, participation would not be in the best interest of the participant (e.g., compromise the well-being) or physical limitations that could prevent, limit, or confound the protocol-specified assessments
* Major surgery (e.g., requiring general anesthesia) ≤4 weeks prior to initiation of study treatment
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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OncoVerity, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Banner MD Anderson

Gilbert, Arizona, United States

Site Status RECRUITING

City of Hope

Duarte, California, United States

Site Status RECRUITING

University of California Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

University of Colorado Health - Anschutz Cancer Pavilion - Anschutz Medical Campus

Aurora, Colorado, United States

Site Status RECRUITING

Yale School of Medicine

New Haven, Connecticut, United States

Site Status RECRUITING

AdventHealth Medical Group Blood &amp; Marrow Transplant at Orlando

Orlando, Florida, United States

Site Status RECRUITING

The University of Iowa Hospitals & Clinics

Iowa City, Iowa, United States

Site Status RECRUITING

University of Kentucky Chandler Medical Center

Lexington, Kentucky, United States

Site Status RECRUITING

Norton Healthcare, Inc.

Louisville, Kentucky, United States

Site Status RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status NOT_YET_RECRUITING

Hofstra/Northwell Health

Lake Success, New York, United States

Site Status RECRUITING

Cornell University

New York, New York, United States

Site Status RECRUITING

University of Rochester Medical Center

Rochester, New York, United States

Site Status RECRUITING

Wake Forest North Carolina

Winston-Salem, North Carolina, United States

Site Status NOT_YET_RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status RECRUITING

Fred Hutch Seattle Cancer Care Alliance

Seattle, Washington, United States

Site Status RECRUITING

Medical College of Wisonsin

Milwaukee, Wisconsin, United States

Site Status NOT_YET_RECRUITING

Tom Baker Cancer Center-Alberta Health Services - University of Calgary

Calgary, Alberta, Canada

Site Status RECRUITING

Stollery Children's Hospital-Walter C Mackenzie Health Sciences Centre - University of Alberta

Edmonton, Alberta, Canada

Site Status RECRUITING

Vancouver General Hospital, Gordon and Leslie Diamond Health Care Centre

Vancouver, British Columbia, Canada

Site Status RECRUITING

University of Western Ontario

London, Ontario, Canada

Site Status RECRUITING

The Ottawa Hospital - General Campus

Ottawa, Ontario, Canada

Site Status RECRUITING

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

Site Status RECRUITING

Saskatchewan Cancer Agency - Saskatoon Cancer Centre

Saskatoon, Saskatchewan, Canada

Site Status RECRUITING

Marien Hospital Duesseldorf

Düsseldorf, , Germany

Site Status RECRUITING

Universitaetsklinik Frankfurt

Frankfurt, , Germany

Site Status RECRUITING

Medizinische Hochschule Hanover, Hannover Medical School

Hanover, , Germany

Site Status RECRUITING

Universitaetsklinik um Schleswig-Holstein, UKSH-Campus Kiel

Kiel, , Germany

Site Status RECRUITING

Inselspital Bern

Bern, , Switzerland

Site Status RECRUITING

HFR Fribourg - Hopital Cantonal

Fribourg, , Switzerland

Site Status RECRUITING

Kantonsspital St. Gallen

Sankt Gallen, , Switzerland

Site Status RECRUITING

Countries

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United States Canada Germany Switzerland

Central Contacts

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OncoVerity OncoVerity

Role: CONTACT

8002201674

Facility Contacts

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Amy Tolenada

Role: primary

Ahmed Aribi, M.D.

Role: primary

877-467-3411

Carla Petro

Role: primary

310-794-0242

Cristina Fresquez-Pizarro

Role: primary

Anne Caldwell

Role: primary

475-434-7321

Advent Health Oncology Research

Role: primary

407-303-2090

Michele Pegg

Role: primary

Lauren Cardano

Role: primary

859-218-7295

Rachel Mattingly, MSN

Role: primary

502-899-3366 ext. 19307

Paritah Patel

Role: primary

551-996-8125

Dina Padula

Role: primary

516-734-3579

Penina Stewart

Role: primary

646-962-4528

Victoria O'Flynn

Role: primary

585-276-3372

Jared Hortman

Role: primary

Ryan Donnelly

Role: primary

Oluyemi Sarumi

Role: primary

403-521-3923

Casey Brodie

Role: primary

780-407-6090

Madeleine Cannings

Role: primary

XXXXXXXXXX

Niko Fragis

Role: primary

XXXXXXXXXX

Holly Carr

Role: primary

Wendy Lui

Role: primary

Priyank Chinta Reddy

Role: primary

306-655-0645

Vera Lohrbacher

Role: primary

49 211 4400 2079

Anja Binckebanck

Role: primary

Victoria Minsch

Role: primary

Ann-Katrin Fichte

Role: primary

Rachelle Mader

Role: primary

Anna Efthymiou

Role: primary

41 026 306 23 33

Marcus Schittenhelm, MD

Role: primary

41714941822

Other Identifiers

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OV-AML-1231

Identifier Type: -

Identifier Source: org_study_id

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