Higher-risk Myelodysplastic SyndromesPhase 2NCT06196203
A Study of AK117 in Combination With Azacitidine in Patients With Myelodysplastic Syndromes
A Phase 2 study testing AK117 and Azacitidine in people with Higher-risk Myelodysplastic Syndromes, run by Akeso at 15 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Higher-risk Myelodysplastic Syndromes
- What is being tested?
- AK117 and Azacitidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 15 sitesin 2 countries
- Study dates
- 7 Feb 2024to Jun 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Show all 9Show fewer
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
15 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
15 sites in 2 countries
United States14 sites, 14 recruiting
- Recruiting
Rocky Mountain Cancer Centers
Aurora, Colorado, 80012
- Recruiting
American Oncology Partners, PA (The Center for Cancer and Blood Disorders-Bethesda)
Bethesda, Maryland, 20817
- Recruiting
Gabrail Cancer Center
Canton, Ohio, 44718
- Recruiting
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27514
- Recruiting
MUSC Hollings Cancer Center
Charleston, South Carolina, 29425
- Recruiting
Maryland Oncology-Columbia
Columbia, Maryland, 21044
- Recruiting
Ohio State University
Columbus, Ohio, 43210
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390-9065
Yazan Madanat, yazan.madanat@utsouthwestern.edu
- Recruiting
Oncology Associates of Oregon
Eugene, Oregon, 97401
Luke Fletcher, luke.fletcher@usoncology.com
- Recruiting
UCLA Ronald Reagan Medical Center
Los Angeles, California, 90095
- Recruiting
Yale Cancer Center
New Haven, Connecticut, 06510
- Recruiting
Mid Florida Hematology and Oncology Center
Orange City, Florida, 32763
- Recruiting
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
- Recruiting
Montefiore Einstein Comprehensive Cancer Center
The Bronx, New York, 10467
China1 site, 1 recruiting
- Recruiting
Institute of hematolongy&blood diseases hospital, chinese academy of medical sciences&peking union medical college
Tianjin
Zhijian Xiao
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Multicenter Phase 2 Study of AK117/Placebo in Combination With Azacitidine in Patients With Newly Diagnosed Higher-risk Myelodysplastic Syndromes
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
- Being tested
- AK117, Placebo, Azacitidine
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AK117Drug
AK117 IV injection
PlaceboDrug
Placebo IV injection
AzacitidineDrug
Azacitidine SC injection
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: 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 Receives: Drug: Placebo; Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete remission rate (CRR)
CRR is defined as the proportion of subjects with complete remission (CR) per International Working Group (IWG) 2023 criteria
Measured: Up to approximately 2 years
Secondary outcomes (10)
Overall response rate (ORR)
The proportion of subjects with recorded response per IWG 2023
Measured: Up to approximately 2 years
Time to response (TTR)
Time from the randomization to the first recorded response
Measured: Up to approximately 2 years
Time to CR (TTCR)
Time from the randomization to the first recorded CR
Measured: Up to approximately 2 years
Duration of response (DoR)
Time from the first recorded response until disease relapse or progression or death due to any cause, whichever occurs first
Measured: Up to approximately 2 years
Duration of CR (DoCR)
Time from the first recorded CR until disease relapse or progression or death due to any cause, whichever occurs first
Measured: Up to approximately 2 years
Event-free survival (EFS)
Time from randomization until transformation to AML or death due to any cause, whichever occurs first
Measured: Up to approximately 2 years
Overall survival (OS)
The time from randomization until death due to any cause
Measured: Up to approximately 2 years
Number of subjects with adverse events (AEs)
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
Measured: Up to approximately 2 years
Pharmacokinetic characteristics
Serum concentrations of AK117 in individual subjects at different time points after AK117 administration
Measured: Up to approximately 2 years
Anti-drug antibody (ADA)
Number of subjects with detectable anti-drug antibodies
Measured: Up to approximately 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2024
- Study start7 Feb 2024
- Primary completionJan 2026 (estimated)
- Study completionJun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Higher-risk Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by AkesoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Akeso
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06196203
- Sponsor's protocol ID
- AK117-205
- Status last verified
- Feb 2025
- First submitted
- 24 Dec 2023
- Last updated
- 11 Feb 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Multicenter Phase 2 Study of AK117/Placebo in Combination With Azacitidine in Patients With Newly Diagnosed Higher-risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT06196203
- Acronym / sponsor ID
- AK117-205
- Conditions
- Higher-risk Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- AkesoIndustry (pharma, biotech, devices)
- Study start
- 7 Feb 2024 (actual)
- Primary completion
- Jan 2026 (estimated)
- Study completion
- Jun 2026 (estimated)
- First posted
- 9 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| AK117 (dose 1) in combination with azacitidine | Experimental | Drug: AK117, Drug: AzacitidineSubjects receive AK117 (dose 1) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously |
| AK117 (dose 2) in combination with azacitidine | Experimental | Drug: AK117, Drug: AzacitidineSubjects receive AK117 (dose 2) intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously |
| Placebo in combination with azacitidine | Placebo comparator | Drug: Placebo, Drug: AzacitidineSubjects receive placebo intravenously, in combination with azacitidine (75 mg/m2, D1-7, Q4W) subcutaneously |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AK117AK117 IV injection | Drug | AK117 (dose 1) in combination with azacitidine, AK117 (dose 2) in combination with azacitidine |
| PlaceboPlacebo IV injection | Drug | Placebo in combination with azacitidine |
| AzacitidineAzacitidine SC injection | Drug | AK117 (dose 1) in combination with azacitidine, AK117 (dose 2) in combination with azacitidine, Placebo in combination with azacitidine |
Primary outcome
Complete remission rate (CRR)
Time frame: Up to approximately 2 years
CRR is defined as the proportion of subjects with complete remission (CR) per International Working Group (IWG) 2023 criteria
10 secondary outcomes
Overall response rate (ORR)
Time frame: Up to approximately 2 years
Time to response (TTR)
Time frame: Up to approximately 2 years
Time to CR (TTCR)
Time frame: Up to approximately 2 years
Duration of response (DoR)
Time frame: Up to approximately 2 years
Duration of CR (DoCR)
Time frame: Up to approximately 2 years
Event-free survival (EFS)
Time frame: Up to approximately 2 years
Overall survival (OS)
Time frame: Up to approximately 2 years
Number of subjects with adverse events (AEs)
Time frame: Up to approximately 2 years
Pharmacokinetic characteristics
Time frame: Up to approximately 2 years
Anti-drug antibody (ADA)
Time frame: Up to approximately 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2025.
- Individual participant dataSharing planned: No
Related trials for Higher-risk Myelodysplastic Syndromes
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
RecruitingPhase 1
First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelodysplastic Syndrome (HR-MDS)
R/R AML, R/R HR-MDS
RecruitingPhase 2
Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)
Myelodysplastic Syndrome
Active, not recruitingPhase 1/2
Quizartinib, Decitabine, and Venetoclax in Treating Participants With Untreated or Relapsed Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome
Acute Myeloid Leukemia, Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
15 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AK117 and Azacitidine in people with Higher-risk Myelodysplastic Syndromes, run by Akeso at 15 sites in 2 countries.
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 9 inclusion and 10 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: looking for participants now, as of the sponsor's last update on 11 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, China. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wenting Li, MD ((+86)18116403289, wenting01.li@akesobio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete remission rate (CRR) (time frame: Up to approximately 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 Feb 2025.