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A Study of AK117 in Combination With Azacitidine in Patients With Myelodysplastic Syndromes

Phase 2
Recruiting
Conditions
Higher-risk Myelodysplastic Syndromes
Interventions
Registration Number
NCT06196203
Lead Sponsor
Akeso
Brief Summary

This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study evaluating the efficacy and safety of AK117 or placebo, combined with azacitidine in patients with newly diagnosed higher-risk myelodysplastic syndromes (HR-MDS).

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
90
Inclusion Criteria
  • Age ≥ 18 years old at the time of enrolment.
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2.
  • Expected life expectancy ≥ 3 months.
  • Newly diagnosed HR-MDS, according to the 2016 World Health Organization (WHO) classification with the presence of < 20% blasts in bone marrow or peripheral blood; Overall IPSS-R score ≥ 3.5.
  • Ability to undergo the study-required bone marrow sample collection procedures.
  • Suitable venous access for the study-required blood sampling (i.e., including PK and immunogenicity).
  • Female patients of childbearing age must have negative serum pregnancy test results before randomization or per region-specific guidance documented in the informed consent and a negative urine pregnancy test on the day of first dose prior to dosing.
  • Female patients of childbearing potential having sex with an unsterilized male partner must agree to use a highly effective method of contraception from the beginning of screening until 180 days after the last dose of the study treatment.
  • Unsterilized male patients having sex with a female partner of childbearing potential must agree to use an effective method of contraception from the beginning of screening until 180 days after the last dose of study treatment.
Exclusion Criteria
  • MDS evolving from a pre-existing myeloproliferative neoplasm (MPN), myelodysplastic/myeloproliferative neoplasms (MDS/MPN).
  • Prior treatment with Cluster of Differentiation (CD) 47 or Signal-regulatory protein alpha (SIRPα)-targeting agents.
  • Concurrently participating in another interventional clinical study, unless it is an observational (non-interventional) clinical study or during the follow-up period of an interventional study.
  • Patients who previously diagnosed with another malignancy and have any evidence of residual disease.
  • Known allergy to any component of any study drug; known history of severe hypersensitivity to other monoclonal antibodies.
  • Patients with any psychiatric or social factor which the investigator deems may interfere with the patient's ability to comply with the requirements of the study.
  • Patients with current hypertension with systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg after oral antihypertensive therapy.
  • Patients with known cardiopulmonary disease defined as unstable angina, clinically significant arrhythmia, congestive heart failure (New York Heart Association Class III or IV), decompensated cirrhosis, nephrotic syndrome, uncontrolled metabolic disorders.
  • Patients who are breastfeeding or plans to breastfeed during the study.
  • Other conditions where the investigator considers the patient inappropriate for enrollment.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
AK117 (dose 1) in combination with azacitidineAzacitidineSubjects receive AK117 (dose 1) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously
AK117 (dose 2) in combination with azacitidineAK117Subjects receive AK117 (dose 2) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously
AK117 (dose 1) in combination with azacitidineAK117Subjects receive AK117 (dose 1) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously
Placebo in combination with azacitidinePlaceboSubjects receive placebo intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously
AK117 (dose 2) in combination with azacitidineAzacitidineSubjects receive AK117 (dose 2) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously
Placebo in combination with azacitidineAzacitidineSubjects receive placebo intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously
Primary Outcome Measures
NameTimeMethod
Complete remission rate (CRR)Up to approximately 2 years

CRR is defined as the proportion of subjects with complete remission (CR) per International Working Group (IWG) 2023 criteria

Secondary Outcome Measures
NameTimeMethod
Time to response (TTR)Up to approximately 2 years

Time from the randomization to the first recorded response

Duration of CR (DoCR)Up to approximately 2 years

Time from the first recorded CR until disease relapse or progression or death due to any cause, whichever occurs first

Duration of response (DoR)Up to approximately 2 years

Time from the first recorded response until disease relapse or progression or death due to any cause, whichever occurs first

Overall response rate (ORR)Up to approximately 2 years

The proportion of subjects with recorded response per IWG 2023

Event-free survival (EFS)Up to approximately 2 years

Time from randomization until transformation to AML or death due to any cause, whichever occurs first

Overall survival (OS)Up to approximately 2 years

The time from randomization until death due to any cause

Time to CR (TTCR)Up to approximately 2 years

Time from the randomization to the first recorded CR

Number of subjects with adverse events (AEs)Up to approximately 2 years

An AE is any untoward medical occurrence in a subject, temporally associated with the use of study treatment, whether or not considered related to the study treatment

Pharmacokinetic characteristicsUp to approximately 2 years

Serum concentrations of AK117 in individual subjects at different time points after AK117 administration

Anti-drug antibody (ADA)Up to approximately 2 years

Number of subjects with detectable anti-drug antibodies

Trial Locations

Locations (15)

Mid Florida Hematology and Oncology Center

🇺🇸

Orange City, Florida, United States

UCLA Ronald Reagan Medical Center

🇺🇸

Los Angeles, California, United States

American Oncology Partners, PA (The Center for Cancer and Blood Disorders-Bethesda)

🇺🇸

Bethesda, Maryland, United States

Washington University School of Medicine in St. Louis

🇺🇸

Saint Louis, Missouri, United States

Montefiore Einstein Comprehensive Cancer Center

🇺🇸

Bronx, New York, United States

Ohio State University

🇺🇸

Columbus, Ohio, United States

MUSC Hollings Cancer Center

🇺🇸

Charleston, South Carolina, United States

Institute of hematolongy&blood diseases hospital, chinese academy of medical sciences&peking union medical college

🇨🇳

Tianjin, China

Rocky Mountain Cancer Centers

🇺🇸

Aurora, Colorado, United States

Oncology Associates of Oregon

🇺🇸

Eugene, Oregon, United States

UT Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Yale Cancer Center

🇺🇸

New Haven, Connecticut, United States

Maryland Oncology-Columbia

🇺🇸

Columbia, Maryland, United States

UNC Lineberger Comprehensive Cancer Center

🇺🇸

Chapel Hill, North Carolina, United States

Gabrail Cancer Center

🇺🇸

Canton, Ohio, United States

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