Acute Myeloid Leukemia, ChildhoodEarly Phase 1NCT05519384
Safety and Preliminary Efficacy of JK500 Cell Injection in Relapsed/Refractory Pediatric Acute Myeloid Leukemia
A Early Phase 1 study testing JK500 cell injection，cyclophosphamide，Fludarabine in people with Acute Myeloid Leukemia, Childhood and Relapsed Leukemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This study is a prospective, single-center, single-arm exploratory clinical study, aiming to complete the preliminary clinical observation of 12 children with relapsed/refractory acute myeloid leukemia treated with JK500 cell injection to evaluate the safety of clinical infusion and the initial efficacy of JK500 cell… Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Acute Myeloid Leukemia, Childhood, Relapsed Leukemia, Refractory Leukemia
- What is being tested?
- JK500 cell injection，cyclophosphamide，Fludarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 14 Sep 2022to Dec 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age ≤18, male or female;
- Patients diagnosed as acute myeloid leukemia (AML) according to the revised World Health Organization (WHO) criteria in 2016;
- Patients who failed to achieve CR after two standard-dose induction therapy or had recurrence within six months after CR;
- The subject or the guardian of the subject must fully understand the purpose, nature, method and possible adverse reactions of the test, agree the subject as the subject, and sign the informed consent.
Who cannot join
- Acute promyelocytic leukemia, chronic myelocytic leukemia, acute mixed-cell leukemia or known central nervous system leukemia;
- AML associated with congenital syndromes such as Down syndrome, Fanconi's anemia, Bloom's syndrome, Koch's syndrome, or congenital aplastic anemia;
- The subjects have active virus infection, and during the screening period, the serum virology test is performed, and the human immunodeficiency virus (HIV) antibody is positive, hepatitis B surface antigen or E antigen is positive, hepatitis C antibody is positive, or treponema pallidum antibody is positive;
- Presence of active systemic infection; Participated in a drug trial within the past 4 weeks;
- Patients who suffered from a clinically significant disease within 28 days before receiving the study product or underwent a major surgical operation within 28 days before receiving the study product, or are expected to need major surgery during the trial;
- children with liver and kidney dysfunction, including:
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- Serum creatinine >2× upper limit of normal reference value;
- Serum total bilirubin > 2× upper limit of normal reference value;
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) > 2× upper limit of normal reference values
- Children who have used live attenuated vaccine 4 weeks before administration or plan to use live attenuated vaccine within 6 months after administration;
- Have any other conditions that may cause the subject to be unable to complete the study or present a significant risk to the subject in the opinion of the Investigator.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Pediatrics, Institute of Hematology and Blood Disease Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
Xiaofan Zhu, MD, 86-21-23909001, xfzhu@ihcams.ac.cn
Min Ruan, MD
About this study
This study is a prospective, single-center, single-arm exploratory clinical study, aiming to complete the preliminary clinical observation of 12 children with relapsed/refractory acute myeloid leukemia treated with JK500 cell injection to evaluate the safety of clinical infusion and the initial efficacy of JK500 cell injection in the treatment of children with relapsed/refractory acute myeloid leukemia.
Read the detailed description
The Main components of JK500 cell injection are regenerative natural killer (NK) cells derived from human embryonic stem cells and 0.9% sodium chloride solution.
- Being tested
- JK500 cell injection，cyclophosphamide，Fludarabine
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JK500 cell injection，cyclophosphamide，FludarabineDrug
After enrollment, patients received JK500 cell injection infusion after reinduction therapy. Pretreatment regimen before cell infusion: intravenous infusion of cyclophosphamide 60mg/kg day -7, intravenous infusion of fludarabine 25mg/㎡/day day -6 to -2 days. In order to activate and expand circulating NK cells, on the day of each infusion of JK500 cells, the patients were first subcutaneously injected with 100WU/ m2 of interleukin (IL)-2 0.5h-1h in advance, for a total of 6 doses.
| Study group | Role | What they receive |
|---|---|---|
| JK500 cell injection | Experimental | They were divided into three dose groups: 10^6, 5x10^6 and 3x10^7 JK500 cells injection /kg/ time, three times a week, a total of 6 times.
According to the principle of dose escalation, 3 patients were assigned to each dose group. After completing the treatment of 3 patients in each dose group, the Safety Review Committee (SRC) discussed whether to enter the next dose group. Receives: Drug: JK500 cell injection，cyclophosphamide，Fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-limiting toxicity (DLT)
DLT evaluation is defined as adverse events or laboratory abnormalities that occur within 4 weeks after investigational drug administration, are unrelated to external causes such as progressive disease, concomitant disease, and concomitant medications, including hematologic and non-hematologic adverse events (grade according to NCI CTCAE 5.0).
Measured: 28 days
Secondary outcomes (2)
Objective Response Rate (ORR)
ORR was defined as complete response (CR)+ incomplete bone marrow recovery (CRi) + partial response (PR). Treatment response will be assessed periodically until the end of treatment and up to 24 months after the first injection.
Measured: 24 months
Minimal Residual Disease (MRD)
To observe the MRD status in bone marrow.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2022
- Study start14 Sep 2022
- Primary completionNov 2023 (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 Acute Myeloid Leukemia, ChildhoodStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- They were divided into three dose groups: 10\^6, 5x10\^6 and 3x10\^7 JK500 cells injection /kg/ time, three times a week, a total of 6 times. According to the principle of dose escalation, 3 patients were assigned to each dose group. After completing the treatment of 3 patients in each dose group, the Safety Review Committee (SRC) discussed whether to enter the next dose group.
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Bejing Institute for Stem Cell and Regenerative Medicine, Institute for Stem cell and Regeneration, Chinese Academy of Sciences
- Responsible party
- Sponsor
- Investigators
- Xiaofan Zhu, Principal investigator, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05519384
- Sponsor's protocol ID
- ISCRNK01001
- Status last verified
- Aug 2022
- First submitted
- 25 Aug 2022
- Last updated
- 28 Nov 2022
- Results posted
- 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 28 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Preliminary Efficacy of JK500 Cell Injection in Relapsed/Refractory Pediatric Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT05519384
- Acronym / sponsor ID
- ISCRNK01001
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Bejing Institute for Stem Cell and Regenerative Medicine, Institute for Stem cell and Regeneration, Chinese Academy of Sciences
- Study start
- 14 Sep 2022 (actual)
- Primary completion
- Nov 2023 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 29 Aug 2022 (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 is a prospective, single-center, single-arm exploratory clinical study, aiming to complete the preliminary clinical observation of 12 children with relapsed/refractory acute myeloid leukemia treated with JK500 cell injection to evaluate the safety of clinical infusion and the initial efficacy of JK500 cell injection in the treatment of children with relapsed/refractory acute myeloid leukemia.
Detailed description
The Main components of JK500 cell injection are regenerative natural killer (NK) cells derived from human embryonic stem cells and 0.9% sodium chloride solution.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- They were divided into three dose groups: 10^6, 5x10^6 and 3x10^7 JK500 cells injection /kg/ time, three times a week, a total of 6 times. According to the principle of dose escalation, 3 patients were assigned to each dose group. After completing the treatment of 3 patients in each dose group, the Safety Review Committee (SRC) discussed whether to enter the next dose group.
| Arm | Type | Receives |
|---|---|---|
| JK500 cell injection | Experimental | Drug: JK500 cell injection，cyclophosphamide，FludarabineThey were divided into three dose groups: 10^6, 5x10^6 and 3x10^7 JK500 cells injection /kg/ time, three times a week, a total of 6 times. According to the principle of dose escalation, 3 patients were assigned to each dose group. After completing the treatment of 3 patients in each dose group, the Safety Review Committee (SRC) discussed whether to enter the next dose group. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Age ≤18, male or female;
- Patients diagnosed as acute myeloid leukemia (AML) according to the revised World Health Organization (WHO) criteria in 2016;
- Patients who failed to achieve CR after two standard-dose induction therapy or had recurrence within six months after CR;
- The subject or the guardian of the subject must fully understand the purpose, nature, method and possible adverse reactions of the test, agree the subject as the subject, and sign the informed consent.
Key exclusion criteria (11 listed)
- Acute promyelocytic leukemia, chronic myelocytic leukemia, acute mixed-cell leukemia or known central nervous system leukemia;
- AML associated with congenital syndromes such as Down syndrome, Fanconi's anemia, Bloom's syndrome, Koch's syndrome, or congenital aplastic anemia;
- The subjects have active virus infection, and during the screening period, the serum virology test is performed, and the human immunodeficiency virus (HIV) antibody is positive, hepatitis B surface antigen or E antigen is positive, hepatitis C antibody is positive, or treponema pallidum antibody is positive;
- Presence of active systemic infection; Participated in a drug trial within the past 4 weeks;
- Patients who suffered from a clinically significant disease within 28 days before receiving the study product or underwent a major surgical operation within 28 days before receiving the study product, or are expected to need major surgery during the trial;
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 |
|---|---|---|
| JK500 cell injection，cyclophosphamide，FludarabineAfter enrollment, patients received JK500 cell injection infusion after reinduction therapy. Pretreatment regimen before cell infusion: intravenous infusion of cyclophosphamide 60mg/kg day -7, intravenous infusion of fludarabine 25mg/㎡/day day -6 to -2 days. In order to activate and expand circulating NK cells, on the day of each infusion of JK500 cells, the patients were first subcutaneously injected with 100WU/ m2 of interleukin (IL)-2 0.5h-1h in advance, for a total of 6 doses. | Drug | JK500 cell injection |
Primary outcome
Dose-limiting toxicity (DLT)
Time frame: 28 days
DLT evaluation is defined as adverse events or laboratory abnormalities that occur within 4 weeks after investigational drug administration, are unrelated to external causes such as progressive disease, concomitant disease, and concomitant medications, including hematologic and non-hematologic adverse events (grade according to NCI CTCAE 5.0).
2 secondary outcomes
Objective Response Rate (ORR)
Time frame: 24 months
Minimal Residual Disease (MRD)
Time frame: 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2022.
- Individual participant dataSharing planned: No
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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.
Xiaofan Zhu principal investigator
Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC
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 JK500 cell injection，cyclophosphamide，Fludarabine in people with Acute Myeloid Leukemia, Childhood and Relapsed Leukemia, run by Institute of Hematology & Blood Diseases Hospital, China 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 4 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: status not confirmed, as of the sponsor's last update on 28 Nov 2022. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Min Ruan, MD (+8613602177144, ruanmin@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose-limiting toxicity (DLT) (time frame: 28 days). 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 28 Nov 2022.