Myelodysplastic SyndromePhase 1/2NCT04900350
A Trial of AK117 (Anti-CD47) in Patients With Myelodysplastic Syndrome
A Phase 1/2 study testing AK117 and Azacitidine in people with Myelodysplastic Syndrome, run by Akeso at 1 site in China.
Trial summary
This is a open label, phase I/II study. All patients are diagnosed with higher-risk MDS, Eastern Cooperative Oncology Group (ECOG) performance status 0-2. The purpose of this study is to evaluate the safety and efficacy of AK117 + azacitidine in subjects with higher-risk MDS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- AK117 and Azacitidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 136 participants
- Where
- 1 siteChina
- Study dates
- 18 Jun 2021to Dec 2025
- Phase
- Phase 1/2
- 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 (at the time consent is obtained).
- Be able and willing to provide written informed consent and to comply with all requirements of study participation (including all study procedures).
- Has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2.
- Has a life expectancy of at least 12 weeks.
- Phase 1: Diagnosis of higher-risk MDS that is either previously untreated or relapsed/refractory.
- Phase 2: Diagnosis of higher-risk MDS that is previously untreated.
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- White blood cell count ≤ 25 x 10^9/L (hydroxyurea may be used to reduce the WBC count).
- Has adequate organ function.
- All female and male subjects of reproductive potential must agree to use an effective method of contraception, as determined by the Investigator, during and for 120 days after the last dose of study treatment.
Who cannot join
- Is currently participating in a study of an investigational agent or using an investigational device.
- Has undergone major surgery within 30 days of Study Day 1.
- Has a known additional malignancy that is progressing or requires systemic treatment. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
- Previous allogeneic hematopoietic stem cell transplant (allo-HSCT) .
- Prior treatment with any anti-CD47 or anti-SIRPα (signal regulatory protein alpha) agent.
- Has an active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
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- Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
- History of myocardial infarction, unstable angina, congestive heart failure within 12 months prior to day 1 of study treatment.
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or is not in the best interest of this subject to participate, in the opinion of the treating investigator.
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the study.
- Has received a live virus vaccine within 30 days of the planned first dose of study therapy.
- Is pregnant, breastfeeding, or expecting to conceive or father a child within the projected duration of the study including 120 days following the last dose of study treatment.
- Has any concurrent medical condition that, in the opinion of the Investigator, would complicate or compromise compliance with the study or the well-being of the subject.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
About this study
Official title: A Phase I/II Trial of AK117 (Anti-CD47) in Patients With Higher-risk Myelodysplastic Syndrome
This is a open label, phase I/II study. All patients are diagnosed with higher-risk MDS, Eastern Cooperative Oncology Group (ECOG) performance status 0-2. The purpose of this study is to evaluate the safety and efficacy of AK117 + azacitidine in subjects with higher-risk MDS.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- AK117, Azacitidine
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AK117Drug
Subjects receive AK117 intravenously.
AzacitidineDrug
Subjects receive Azacitidine subcutaneously.
| Study group | Role | What they receive |
|---|---|---|
| AK117+ azacitidine | Experimental | Phase 1: Subjects will receive escalating doses of A117 in combination with azacitidine 75 mg/m2 subcutaneous daily for 7 days of a 28 day cycle;
Phase 2: Subjects will receive AK117 at the recommended Phase 2 dose in combination with azacitidine 75 mg/m2 subcutaneous daily for 7 days of a 28-day cycle. Receives: Drug: AK117; Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Complete Remission (CR)
Number of participants achieving a CR per International Working Group (IWG) 2006 criteria.
Measured: Approximately 6 months
Number of participants with adverse events (AEs)
An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product temporally associated with the use of study treatment, whether or not considered related to the study treatment.
Measured: Up to approximately 2 years.
Duration of Complete Remission (DoCR)
The DoCR is measured from the time measurement criteria are first met for CR to the first date of relapse or death, whichever occurs earlier.
Measured: Up to approximately 2 years.
Secondary outcomes (5)
Objective Response Rate (ORR)
ORR is defined as the proportion of participants who reach objective response including CR, Partial Remission (PR), marrow CR, or hematologic improvement per IWG 2006 MDS criteria prior to initiation of any new MDS therapy.
Measured: Up to approximately 2 years.
Duration of Response (DOR)
DOR is measured from the time measurement criteria are first met for objective response to the first date of relapse or death, whichever occurs earlier.
Measured: Up to approximately 2 years.
Progression Free Survival (PFS)
PFS is defined as the time from the start of treatment with AK117 until the first documentation of disease progression or death due to any cause, whichever occurs first.
Measured: Up to approximately 2 years.
Maximum observed concentration (Cmax) of AK117
Serum concentrations of AK117 in individual subjects at different time points after AK117 administration
Measured: Up to 2 years.
Anti-drug antibodies (ADA)
Number of subjects with detectable anti-drug antibodies (ADA).
Measured: Up to 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2021
- Study start18 Jun 2021
- Primary completionSep 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 136 participants (actual)
Sponsor and investigators
- Lead sponsor
- Akeso
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Depei Wu, MD, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04900350
- Sponsor's protocol ID
- AK117-103
- Status last verified
- Feb 2025
- First submitted
- 19 May 2021
- Last updated
- 3 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Trial of AK117 (Anti-CD47) in Patients With Higher-risk Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT04900350
- Acronym / sponsor ID
- AK117-103
- Conditions
- Myelodysplastic Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 136 participants (actual)
- Lead sponsor
- AkesoIndustry (pharma, biotech, devices)
- Study start
- 18 Jun 2021 (actual)
- Primary completion
- Sep 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 25 May 2021 (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 open label, phase I/II study. All patients are diagnosed with higher-risk MDS, Eastern Cooperative Oncology Group (ECOG) performance status 0-2. The purpose of this study is to evaluate the safety and efficacy of AK117 + azacitidine in subjects with higher-risk MDS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AK117+ azacitidine | Experimental | Drug: AK117, Drug: AzacitidinePhase 1: Subjects will receive escalating doses of A117 in combination with azacitidine 75 mg/m2 subcutaneous daily for 7 days of a 28 day cycle; Phase 2: Subjects will receive AK117 at the recommended Phase 2 dose in combination with azacitidine 75 mg/m2 subcutaneous daily for 7 days of a 28-day cycle. |
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 (at the time consent is obtained).
- Be able and willing to provide written informed consent and to comply with all requirements of study participation (including all study procedures).
- Has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2.
- Has a life expectancy of at least 12 weeks.
- Phase 1: Diagnosis of higher-risk MDS that is either previously untreated or relapsed/refractory.
Key exclusion criteria (13 listed)
- Is currently participating in a study of an investigational agent or using an investigational device.
- Has undergone major surgery within 30 days of Study Day 1.
- Has a known additional malignancy that is progressing or requires systemic treatment. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
- Previous allogeneic hematopoietic stem cell transplant (allo-HSCT) .
- Prior treatment with any anti-CD47 or anti-SIRPα (signal regulatory protein alpha) agent.
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 |
|---|---|---|
| AK117Subjects receive AK117 intravenously. | Drug | AK117+ azacitidine |
| AzacitidineSubjects receive Azacitidine subcutaneously. | Drug | AK117+ azacitidine |
Primary outcome
Rate of Complete Remission (CR)
Time frame: Approximately 6 months
Number of participants achieving a CR per International Working Group (IWG) 2006 criteria.
Number of participants with adverse events (AEs)
Time frame: Up to approximately 2 years.
An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product temporally associated with the use of study treatment, whether or not considered related to the study treatment.
Duration of Complete Remission (DoCR)
Time frame: Up to approximately 2 years.
The DoCR is measured from the time measurement criteria are first met for CR to the first date of relapse or death, whichever occurs earlier.
5 secondary outcomes
Objective Response Rate (ORR)
Time frame: Up to approximately 2 years.
Duration of Response (DOR)
Time frame: Up to approximately 2 years.
Progression Free Survival (PFS)
Time frame: Up to approximately 2 years.
Maximum observed concentration (Cmax) of AK117
Time frame: Up to 2 years.
Anti-drug antibodies (ADA)
Time frame: Up to 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 3 Mar 2025.
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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. This study is not recruiting at the moment.
Depei Wu, MD principal investigator
The First Affiliated Hospital of Soochow University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AK117 and Azacitidine in people with Myelodysplastic Syndrome, run by Akeso at 1 site in China.
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 13 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: under way, not looking for participants, as of the sponsor's last update on 3 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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?
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?
Rate of Complete Remission (CR) (time frame: Approximately 6 months). 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?
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 3 Mar 2025.