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

NCT06196203 | Recruiting Phase 2 FDA Drug

• 1 other identifier: AK117-205
• Study Type: interventional
• Target: 90
• Locations: 2 countries
• Sites: 15

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).

Conditions

Higher-risk Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• Complete remission rate (CRR)

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

  Up to approximately 2 years

Secondary Outcomes (10)

• Overall response rate (ORR)

  Up to approximately 2 years

• Time to response (TTR)

  Up to approximately 2 years

• Time to CR (TTCR)

  Up to approximately 2 years

• Duration of response (DoR)

  Up to approximately 2 years

• Duration of CR (DoCR)

  Up to approximately 2 years

Study Arms (3)

AK117 (dose 1) in combination with azacitidine

EXPERIMENTAL

Subjects receive AK117 (dose 1) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously

Drug: AK117; Drug: Azacitidine

AK117 (dose 2) in combination with azacitidine

EXPERIMENTAL

Subjects receive AK117 (dose 2) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously

Drug: AK117; Drug: Azacitidine

Placebo in combination with azacitidine

PLACEBO COMPARATOR

Subjects receive placebo intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously

Drug: Placebo; Drug: Azacitidine

Interventions

AK117 DRUG

AK117 IV injection

AK117 (dose 1) in combination with azacitidine; AK117 (dose 2) in combination with azacitidine

Placebo DRUG

Placebo IV injection

Placebo in combination with azacitidine

Azacitidine DRUG

Azacitidine SC injection

AK117 (dose 1) in combination with azacitidine; AK117 (dose 2) in combination with azacitidine; Placebo in combination with azacitidine

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Akeso: lead

Study Sites (15)

UCLA Ronald Reagan Medical Center

Los Angeles, California, 90095, United States

RECRUITING

Rocky Mountain Cancer Centers

Aurora, Colorado, 80012, United States

RECRUITING

Yale Cancer Center

New Haven, Connecticut, 06510, United States

RECRUITING

Mid Florida Hematology and Oncology Center

Orange City, Florida, 32763, United States

RECRUITING

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

Bethesda, Maryland, 20817, United States

RECRUITING

Maryland Oncology-Columbia

Columbia, Maryland, 21044, United States

RECRUITING

Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

RECRUITING

Montefiore Einstein Comprehensive Cancer Center

The Bronx, New York, 10467, United States

RECRUITING

UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27514, United States

RECRUITING

Gabrail Cancer Center

Canton, Ohio, 44718, United States

RECRUITING

Ohio State University

Columbus, Ohio, 43210, United States

RECRUITING

Oncology Associates of Oregon

Eugene, Oregon, 97401, United States

RECRUITING

MUSC Hollings Cancer Center

Charleston, South Carolina, 29425, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390-9065, United States

RECRUITING

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

Tianjin, China

RECRUITING

MeSH Terms

Interventions

Azacitidine

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Central Study Contacts

Wenting Li, MD

CONTACT

(+86)18116403289; wenting01.li@akesobio.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 24, 2023
• First Posted: January 9, 2024
• Study Start: February 7, 2024
• Primary Completion: January 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: February 11, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (14); CN (1)