Antisynthetase SyndromeEarly Phase 1NCT06613490
An Exploratory Clinical Study of CD19 CAR NK Cells for the Treatment of Refractory Antisynthetase Antibody Syndrome and Rheumatoid Arthritis
A Early Phase 1 study testing anti CD19 CAR NK cells in people with Antisynthetase Syndrome and Rheumatoid Arthritis, run by The First Affiliated Hospital with Nanjing Medical University at 1 site in China.
Trial summary
A single-center, open-label dose-escalation design to evaluate the safety and efficacy of 3 infusions of anti CD19 CAR NK cells (KN5501), as well as the expansion and persistence of KN5501 in patients with refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA); To evaluate the ability of… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Antisynthetase Syndrome, Rheumatoid Arthritis
- What is being tested?
- anti CD19 CAR NK cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteChina
- Study dates
- 20 May 2025to 20 Oct 2026
- Phase
- Early Phase 1
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects voluntarily sign the Informed Consent Form (ICF) , participate in this clinical study and be willing to follow and be able to complete all trial procedures.
- Defined according to the 2010 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) classification criteria, adult patients with RA diagnosed ≥3 months prior to screening;Moderately to severely active RA;Poor response, or loss of response, or intolerance to at least one conventional synthetic DMARD (csDMARD) or biologic DMARD (bDMARD) or targeted synthetic DMARD (tsDMARD).
- Defined according to the 2020 ENMC-DM classification diagnostic criteria. Adult patients with ASyS diagnosed ≥3 months prior to screening; patients with moderately severe active ASyS.
- Age: ≥ 18 years old and ≤ 70 years old, male or female.
- Subjects with estimated survival > 12 weeks.
- Serum creatinine clearance meets the relevant age/sex criteria, and aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 times the upper limit of normal (ULN).
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- ECOG score 0 - 2.
- The heart structure is essentially normal by echocardiography and Left ventricular ejection fraction (LVEF) ≥45%.
- 2 weeks after the subject received the last dose treatment (hormonal, immunosuppressive or other experimental treatment).
Who cannot join
- Subjects with known severe allergic reactions, hypersensitivity, contraindication to any medications during the trial (cyclophosphamide, fludarabine, tozumabs), or subjects with a history of severe allergic reactions.
- Subjects with one of the following genetic syndromes: Fanconi syndrome, Kostmann syndrome, Shwachman syndrome or any of the known bone marrow failure syndromes.
- Subjects with Active or uncontrolled infections requiring parenteral antimicrobials; evidence of severe active viral or bacterial infections or uncontrolled systemic fungal infections.
- Subjects with grade III or IV heart failure (NYHA classification).
- History of epilepsy or other central nervous system (CNS) diseases.
- Subjects with history of malignancy except cured of carcinoma in situ of the skin or cervix, and patients with inactive tumors.
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- Subjects with pronounced bleeding tendencies, such as gastrointestinal bleeding, coagulation disorders, and hypersplenism.
- The subject with unstable angina, symptomatic congestive heart failure or myocardial infarction within the last 6 months.
- Females who are pregnant, lactating, or planning a pregnancy within six months.
- Subjects who have received other clinical trial treatment within 3 months.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital with Nanjing Medical University
Nanjing, Jiangsu, 210029
Wenfeng Tan, PhD, MD, 086 18061202878, tanwenfeng@jsph.org.cn
Hanxiao You, PhD, MD, 086 18800181269, youhanxiao@jsph.org.cn
About this study
Official title: An Exploratory Clinical Study of the Safety and Efficacy of CD19 Chimeric Antigen Receptor NK Cells for the Treatment of Refractory Antisynthetase Antibody Syndrome and Rheumatoid Arthritis
A single-center, open-label dose-escalation design to evaluate the safety and efficacy of 3 infusions of anti CD19 CAR NK cells (KN5501), as well as the expansion and persistence of KN5501 in patients with refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA); To evaluate the ability of KN5501 to clear CD19-positive B cells in patients to determine the feasibility of KN5501 for the treatment of refractory ASyS and or RA.
- Conditions
- Antisynthetase Syndrome, Rheumatoid Arthritis
- Being tested
- anti CD19 CAR NK cells
- Medical subject headings
- Antisynthetase syndrome, Arthritis, Rheumatoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti CD19 CAR NK cellsBiological
This study is a single-arm, open-label and single-center exploratory clinical study to evaluate the safety and effectiveness of anti CD19 CAR NK cells in patients with refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA). All subjects will receive fludarabine/cyclophosphamide lymphodepletion followed by Anti-CD19 CAR NK cells infusion.
| Study group | Role | What they receive |
|---|---|---|
| anti CD19 CAR NK cells | Experimental | Not described Receives: Biological: anti CD19 CAR NK cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Dose-Limiting Toxicity (DLT)
To characterize the safety of CD19 CAR NK Cells (KN5501) for refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA)
Measured: up to 52 weeks after infusion
Incidence of Treatment Emergent Adverse Events (TEAEs)
To characterize the safety of CD19 CAR NK Cells (KN5501) for refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA)
Measured: up to 52 weeks after infusion
Secondary outcomes (1)
The overall response rate (ORR)
To characterize the efficacy of CD19 CAR NK Cell (KN5501) for refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA)
Measured: 4, 12, 24, 36 and 52 months after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2024
- Study start20 May 2025 (estimated)
- Primary completion20 Oct 2025 (estimated)
- Study completion20 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Antisynthetase SyndromeStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital with Nanjing Medical UniversityThe 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
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Rui Therapeutics Co., Ltd
- Responsible party
- Principal investigator, Wenfeng Tan, The First Affiliated Hospital with Nanjing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06613490
- Sponsor's protocol ID
- 2023-SR-834
- Status last verified
- Apr 2025
- First submitted
- 23 Sep 2024
- Last updated
- 30 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Clinical Study of the Safety and Efficacy of CD19 Chimeric Antigen Receptor NK Cells for the Treatment of Refractory Antisynthetase Antibody Syndrome and Rheumatoid Arthritis
- ClinicalTrials.gov ID
- NCT06613490
- Acronym / sponsor ID
- 2023-SR-834
- Conditions
- Antisynthetase Syndrome, Rheumatoid Arthritis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital with Nanjing Medical UniversityUniversity, hospital or other
- Collaborators
- Rui Therapeutics Co., Ltd
- Study start
- 20 May 2025 (estimated)
- Primary completion
- 20 Oct 2025 (estimated)
- Study completion
- 20 Oct 2026 (estimated)
- First posted
- 26 Sep 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.
A single-center, open-label dose-escalation design to evaluate the safety and efficacy of 3 infusions of anti CD19 CAR NK cells (KN5501), as well as the expansion and persistence of KN5501 in patients with refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA); To evaluate the ability of KN5501 to clear CD19-positive B cells in patients to determine the feasibility of KN5501 for the treatment of refractory ASyS and or RA.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| anti CD19 CAR NK cells | Experimental | Biological: anti CD19 CAR NK cells |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subjects voluntarily sign the Informed Consent Form (ICF) , participate in this clinical study and be willing to follow and be able to complete all trial procedures.
- Defined according to the 2010 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) classification criteria, adult patients with RA diagnosed ≥3 months prior to screening;Moderately to severely active RA;Poor response, or loss of response, or intolerance to at least one conventional synthetic DMARD (csDMARD) or biologic DMARD (bDMARD) or targeted synthetic DMARD (tsDMARD).
- Defined according to the 2020 ENMC-DM classification diagnostic criteria. Adult patients with ASyS diagnosed ≥3 months prior to screening; patients with moderately severe active ASyS.
- Age: ≥ 18 years old and ≤ 70 years old, male or female.
- Subjects with estimated survival > 12 weeks.
Key exclusion criteria (10 listed)
- Subjects with known severe allergic reactions, hypersensitivity, contraindication to any medications during the trial (cyclophosphamide, fludarabine, tozumabs), or subjects with a history of severe allergic reactions.
- Subjects with one of the following genetic syndromes: Fanconi syndrome, Kostmann syndrome, Shwachman syndrome or any of the known bone marrow failure syndromes.
- Subjects with Active or uncontrolled infections requiring parenteral antimicrobials; evidence of severe active viral or bacterial infections or uncontrolled systemic fungal infections.
- Subjects with grade III or IV heart failure (NYHA classification).
- History of epilepsy or other central nervous system (CNS) diseases.
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 |
|---|---|---|
| anti CD19 CAR NK cellsThis study is a single-arm, open-label and single-center exploratory clinical study to evaluate the safety and effectiveness of anti CD19 CAR NK cells in patients with refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA). All subjects will receive fludarabine/cyclophosphamide lymphodepletion followed by Anti-CD19 CAR NK cells infusion. | Biological | anti CD19 CAR NK cells |
Primary outcome
Incidence of Dose-Limiting Toxicity (DLT)
Time frame: up to 52 weeks after infusion
To characterize the safety of CD19 CAR NK Cells (KN5501) for refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA)
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: up to 52 weeks after infusion
To characterize the safety of CD19 CAR NK Cells (KN5501) for refractory antisynthetase antibody syndrome (ASyS) and rheumatoid arthritis (RA)
1 secondary outcome
The overall response rate (ORR)
Time frame: 4, 12, 24, 36 and 52 months after infusion
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 30 Apr 2025.
- 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.
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 anti CD19 CAR NK cells in people with Antisynthetase Syndrome and Rheumatoid Arthritis, run by The First Affiliated Hospital with Nanjing Medical University 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 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 30 Apr 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 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?
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 Wenfeng Tan, PhD, MD (086 18061202878, tanwenfeng@jsph.org.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Dose-Limiting Toxicity (DLT) (time frame: up to 52 weeks after infusion). 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 Apr 2025.