Relapsed/Refractory Acute Myeloid LeukemiaEarly Phase 1NCT06541405
Safety and Efficacy of NK520 to Treat Pediatric Relapsed/Refractory Acute Myeloid Leukemia
A Early Phase 1 study testing NK520 in people with Relapsed/Refractory Acute Myeloid Leukemia, run by Base Therapeutics (Shanghai) Co., Ltd. at 1 site in China.
Trial summary
This study will evaluate the safety and efficacy of NK520 in the treatment of pediatric relapsed/refractory acute myeloid leukemia. NK520 will be administered by intravenous injection. The safety and efficacy of this treatment will be evaluated. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Relapsed/Refractory Acute Myeloid Leukemia
- What is being tested?
- NK520
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2024to 1 Jan 2025
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must be between 6 and 18 years;
- Diagnostic Criteria: Meet the 2022 World Health Organization (WHO) diagnostic criteria for acute myelocytic leukemia (AML), unsuitable for current treatments or patients with relapsed/refractory AML after ≥2 lines of therapy. The definition of relapsed/refractory acute myeloid leukemia is based on the 2017 Chinese Guidelines for Diagnosis and Treatment: a. Relapsed AML: Diagnosis is confirmed when leukemia cells reappear in the peripheral blood or bone marrow blast cells exceed 5% after complete remission (CR) (excluding reasons such as bone marrow regeneration post-consolidation chemotherapy) or there is extramedullary infiltration by leukemia cells; b. Refractory AML: Initial cases unresponsive after two cycles of standard regimen treatment; recurrence within 12 months after CR and consolidation therapy; recurrence beyond 12 months with ineffectiveness of conventional chemotherapy; those who have relapsed twice or more; or persistent extramedullary leukemia;
- For participants under 16 years old, Lansky performance status must be ≥50%; for participants aged 16 or older, Karnofsky performance status must be ≥50%;
- Expected survival of at least 12 weeks;
- Normal Organ Function.
Who cannot join
- Acute promyelocytic leukemia, chronic myeloid leukemia, acute mixed lineage leukemia, or known central nervous system leukemia;
- AML associated with congenital syndromes, such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome, or congenital aplastic anemia;
- Severe bleeding tendency or coagulation disorders, or currently receiving thrombolytic therapy;
- Human immunodeficiency virus (HIV)-infected individuals, or individuals with known active syphilis infection;
- Receipt of live attenuated vaccines within 2 weeks before the first dose or planned during the study period;
- Participation in another clinical trial and receipt of investigational drug within 4 weeks prior to the first dose;
Show all 11Show fewer
- Receipt of immune-modulatory drugs (including thymosin, interferons, except for local use to manage conditions like pleural or ascites fluid) within 2 weeks before the first dose;
- At screening, positive hepatitis B or C viral markers as follows:
- Hepatitis B surface antigen (HBsAg) positive with serum hepatitis B virus-deoxyribonucleic acid (HBV-DNA) level ≥1×10^3 copies/mL or above normal range;
- Positive for hepatitis C virus (HCV) antibodies;
- Any other condition or situation in which the investigator deems the patient unsuitable for participation in this study.
Trial locations and contacts
1 site in 1 country.
China1 site
Shanghai Children Medical Center
Shanghai, Shanghai Municipality, 200127
About this study
Official title: An Open, Single Center Exploratory Study to Evaluate Safety and Efficacy of NK520 for Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia
This study will evaluate the safety and efficacy of NK520 in the treatment of pediatric relapsed/refractory acute myeloid leukemia. NK520 will be administered by intravenous injection. The safety and efficacy of this treatment will be evaluated.
Read the detailed description
This open label, single-arm study aims to evaluate the efficacy and safety of allogenic natural killer (NK) cells in pediatric relapsed/refractory acute myeloid leukemia. Allogenic NK cells will be infused once a week. After infusion, the investigators will observe the characteristics of dose limited toxicity (DLT), and determine the maximum tolerable dose(MTD). To provide basis for the dosage and treatment plan of cell products in follow-up clinical trials.
- Being tested
- NK520
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NK520Drug
Also called genetic modified NK cell
The number of NK520 cell infused for each dosing should be calculated base on the body weight of subject. NK520 will be administered through intravenous infusion.
| Study group | Role | What they receive |
|---|---|---|
| Group A(low-dose group) | Experimental | NK520: 5x10^7 NK/kg Receives: Drug: NK520 |
| Group B(medium-dose group) | Experimental | NK520: 1.5×10^8NK/kg Receives: Drug: NK520 |
| Group C(high-dose group) | Experimental | NK520: 3×10^8NK/kg Receives: Drug: NK520 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-Limiting Toxicity
To evaluate the DLT during N520 treatment
Measured: From the first infusion of NK520 to 4 weeks after last infusion of NK520
Complete Response Rate (CRR)
Effectiveness Metrics
Measured: from the date of first infusion of NK510 up to 104 weeks
Secondary outcomes (4)
Overall response rate (ORR)
Effectiveness Metrics
Measured: from the date of first infusion of NK510 up to 104 weeks
Duration of Response (DOR)
Effectiveness Metrics
Measured: From the date of enrollment up to 104 weeks
Event-Free Survival (EFS)
Effectiveness Metrics
Measured: From date of enrollment up to 104 weeks, or progression, or date of death, whichever came first.
Overall Survival (OS)
Effectiveness Metrics
Measured: From date of enrollment up to 104 weeks, or date of death, whichever came first.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2024
- Study start1 Jul 2024
- Primary completion1 Jan 2025
- Study completion1 Jan 2025
Why it stopped: poor accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Dose escalation study
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Base Therapeutics (Shanghai) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Shanghai Children's Medical Center
- Responsible party
- Sponsor
- Investigators
- wenting Hu, Principal investigator, Shanghai children's medical center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06541405
- Sponsor's protocol ID
- NK520-01
- Status last verified
- Sep 2026
- First submitted
- 7 Jun 2024
- Last updated
- 30 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open, Single Center Exploratory Study to Evaluate Safety and Efficacy of NK520 for Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT06541405
- Acronym / sponsor ID
- NK520-01
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: poor accrual
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Base Therapeutics (Shanghai) Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Shanghai Children's Medical Center
- Study start
- 1 Jul 2024 (actual)
- Primary completion
- 1 Jan 2025 (actual)
- Study completion
- 1 Jan 2025 (actual)
- First posted
- 7 Aug 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 study will evaluate the safety and efficacy of NK520 in the treatment of pediatric relapsed/refractory acute myeloid leukemia. NK520 will be administered by intravenous injection. The safety and efficacy of this treatment will be evaluated.
Detailed description
This open label, single-arm study aims to evaluate the efficacy and safety of allogenic natural killer (NK) cells in pediatric relapsed/refractory acute myeloid leukemia. Allogenic NK cells will be infused once a week. After infusion, the investigators will observe the characteristics of dose limited toxicity (DLT), and determine the maximum tolerable dose(MTD). To provide basis for the dosage and treatment plan of cell products in follow-up clinical trials.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Dose escalation study
| Arm | Type | Receives |
|---|---|---|
| Group A(low-dose group) | Experimental | Drug: NK520NK520: 5x10^7 NK/kg |
| Group B(medium-dose group) | Experimental | Drug: NK520NK520: 1.5×10^8NK/kg |
| Group C(high-dose group) | Experimental | Drug: NK520NK520: 3×10^8NK/kg |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Participants must be between 6 and 18 years;
- Diagnostic Criteria: Meet the 2022 World Health Organization (WHO) diagnostic criteria for acute myelocytic leukemia (AML), unsuitable for current treatments or patients with relapsed/refractory AML after ≥2 lines of therapy. The definition of relapsed/refractory acute myeloid leukemia is based on the 2017 Chinese Guidelines for Diagnosis and Treatment: a. Relapsed AML: Diagnosis is confirmed when leukemia cells reappear in the peripheral blood or bone marrow blast cells exceed 5% after complete remission (CR) (excluding reasons such as bone marrow regeneration post-consolidation chemotherapy) or there is extramedullary infiltration by leukemia cells; b. Refractory AML: Initial cases unresponsive after two cycles of standard regimen treatment; recurrence within 12 months after CR and consolidation therapy; recurrence beyond 12 months with ineffectiveness of conventional chemotherapy; those who have relapsed twice or more; or persistent extramedullary leukemia;
- For participants under 16 years old, Lansky performance status must be ≥50%; for participants aged 16 or older, Karnofsky performance status must be ≥50%;
- Expected survival of at least 12 weeks;
- Normal Organ Function.
Key exclusion criteria (11 listed)
- Acute promyelocytic leukemia, chronic myeloid leukemia, acute mixed lineage leukemia, or known central nervous system leukemia;
- AML associated with congenital syndromes, such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome, or congenital aplastic anemia;
- Severe bleeding tendency or coagulation disorders, or currently receiving thrombolytic therapy;
- Human immunodeficiency virus (HIV)-infected individuals, or individuals with known active syphilis infection;
- Receipt of live attenuated vaccines within 2 weeks before the first dose or planned during the study period;
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 |
|---|---|---|
| NK520Also: genetic modified NK cellThe number of NK520 cell infused for each dosing should be calculated base on the body weight of subject. NK520 will be administered through intravenous infusion. | Drug | Group A(low-dose group), Group B(medium-dose group), Group C(high-dose group) |
Primary outcome
Dose-Limiting Toxicity
Time frame: From the first infusion of NK520 to 4 weeks after last infusion of NK520
To evaluate the DLT during N520 treatment
Complete Response Rate (CRR)
Time frame: from the date of first infusion of NK510 up to 104 weeks
Effectiveness Metrics
4 secondary outcomes
Overall response rate (ORR)
Time frame: from the date of first infusion of NK510 up to 104 weeks
Duration of Response (DOR)
Time frame: From the date of enrollment up to 104 weeks
Event-Free Survival (EFS)
Time frame: From date of enrollment up to 104 weeks, or progression, or date of death, whichever came first.
Overall Survival (OS)
Time frame: From date of enrollment up to 104 weeks, or date of death, whichever came first.
Results and safety
The study was stopped early (reason given: poor accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Relapsed/Refractory Acute Myeloid Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
Not yet recruitingPhase 1
A Study to Assess the Pharmacokinetics and Safety of Revumenib in Participants With Advanced Cancer and Hepatic or Renal Impairment
Relapsed/Refractory (R/R) Hematologic Malignancies, Acute Leukemia
Active, not recruitingPhase 3
Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)
Acute Myeloid Leukemia
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
Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax
IDH1 Mutation, Relapsed / Refractory AML
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.
wenting Hu principal investigator
Shanghai children's medical center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing NK520 in people with Relapsed/Refractory Acute Myeloid Leukemia, run by Base Therapeutics (Shanghai) Co., Ltd. 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 5 inclusion and 11 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: stopped early, as of the sponsor's last update on 30 Sep 2026. 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: poor accrual). Results are not available in the source record.
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?
Dose-Limiting Toxicity (time frame: From the first infusion of NK520 to 4 weeks after last infusion of NK520). The study lists 2 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 30 Sep 2026.