Thrombocytopenia AlloimmuneEarly Phase 1NCT06337474
An Clinical Study of CD19 CAR NK Cells for the Treatment of Refractory Primary Immune Thrombocytopenia
A Early Phase 1 study testing anti-CD19 CAR NK cells (KN5501) in people with Thrombocytopenia Alloimmune, run by Changzhou No.2 People's Hospital at 1 site in China.
Trial summary
A single arm, open-label pilot study is designed to determine the safety and effectiveness of CD19 CAR NK cells (KN5501) in patients with refractory immune thrombocytopenia. 9 patients are planned to be enrolled in the dose-escalation trial (9×10^9 cells, 13.5×10^9 cells). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Thrombocytopenia Alloimmune
- What is being tested?
- anti-CD19 CAR NK cells (KN5501)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 30 Aug 2024to 30 Jul 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age: ≥ 18 years old and ≤ 65 years old, male or female, subjects voluntarily participate in this clinical study and sign the Informed Consent Form (ICF);
- Diagnosis of immune thrombocytopenia (UTP) (according to American Society of Hematology 2019 guidelines and International ITP Working Group);
- Platelet count is decreased in routine blood tests at least two consecutive times, with no obvious abnormalities in the morphology of blood cells on microscopic examination of peripheral blood smears;
- Spleen is not enlarged during screening;
- The morphology of bone marrow cells in subjects is characterized by increased or normal megakaryocytes with impaired maturation;
- Exclude other secondary thrombocytopenia: secondary thrombocytopenia due to autoimmune diseases, thyroid disorders, lymphoproliferative disorders, myelodysplastic syndromes (MDS), aplastic anemia (AA), various malignant blood disorders, neoplastic infiltrates, chronic liver disease, hypersplenism, common variable immunodeficiency disease (CVID), infections, vaccinations, etc.; thrombocytopenia; drug-induced thrombocytopenia; homozygous thrombocytopenia; thrombocytopenia during pregnancy; congenital thrombocytopenia and pseudothrombocytopenia. Thrombocytopenia due to consumption; drug-induced thrombocytopenia; isoimmune thrombocytopenia; thrombocytopenia in pregnancy; congenital thrombocytopenia and pseudothrombocytopenia;
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- Subjects with refractory ITP who are refractory to first-line therapeutic agents, thrombopoietic agents in second-line therapy, and rituximab, or who have had ineffective splenectomies/postoperative recurrences, and who undergo diagnostic reassessment and remain diagnosed with ITP;
- Patients with refractory ITP: refractory to first-line therapeutic agents, thrombopoietic agents in second-line therapy, and rituximab; patients diagnosed with ITP despite unsuccessful splenectomy/postoperative recurrence on diagnostic reassessment
- ECOG score ≤ 1;
- Left ventricular ejection fraction (LVEF) ≥50% and no clinically significant pericardial effusion;
- ≥ 4 weeks after subjects received last dose treatment (Radiotherapy, chemotherapy, monoclonal antibody therapy or other treatments);
- NRT, antimalarial monotherapy, antimalarials in combination with OCS and/or immunosuppressants, combination of OCS and/or immunosuppressants.
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 active infection receiving intravenous (IV) antibiotic treatment, or received intravenous (IV) antibiotic treatment within one week prior to anti-CD19 CAR NK Cell (KN5501) infusion;
- Subjects with acquired and congenital immunodeficiency diseases;
- Subjects with grade III or IV heart failure (NYHA classification);
- History of epilepsy or other central nervous system (CNS) diseases;
- History of severe herpes infections such as herpes encephalitis, ocular herpes, or diffuse herpes;
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- History of other primary malignant tumors except:
- a Cured non-melanoma skin cancer by surgical excision, for example basal cell carcinoma (BCC) ; b Cured primary malignant tumors, such as cervical cancer, superficial bladder cancer, breast cancer;
- Signs of herpes or varicella zoster virus infection (specifically varicella, zoster) within 12 weeks prior to screening; history of any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urinary, pulmonary, neurologic, dermatologic, psychiatric, and renal disease or other significant medical condition, other than lupus, that prevents administration of BIIB059 (as determined by the investigator)
- Females who are pregnant, lactating, or planning a pregnancy within six months;
- History or current diagnosis of clinically significant non-ITP-induced thrombocytopenia
- Subjects who have received other clinical trial treatment within 3 month;
- Any situation judged by the investigators that may increase the risk of the subjects or interfere with the clinical trial outcome.
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
Changzhou Second People's Hospital
Changzhou, Jiangsu, 213000
Wei Qin, doctor, +8618796912763, qinwei19840601@163.com
About this study
Official title: An Exploratory Clinical Study of the Safety and Efficacy of CD19 Chimeric Antigen Receptor NK Cell Injections for the Treatment of Refractory Primary Immune Thrombocytopenia
A single arm, open-label pilot study is designed to determine the safety and effectiveness of CD19 CAR NK cells (KN5501) in patients with refractory immune thrombocytopenia. 9 patients are planned to be enrolled in the dose-escalation trial (9×10^9 cells, 13.5×10^9 cells). The primary objective of the study is to evaluation of the safety and feasibility of KN5501 for the treatment of relapsed/refractory B-cell related autoimmune diseases. The secondary objective is to evaluate evaluation of KN5501 for the treatment of refractory immune thrombocytopenia. The exploratory objective is to evaluate expansion, persistence and ability to deplete CD19 positive B cells of KN5501 in patients with refractory immune thrombocytopenia.
- Conditions
- Thrombocytopenia Alloimmune
- Being tested
- anti-CD19 CAR NK cells (KN5501)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-CD19 CAR NK cells (KN5501)Drug
Patients will receive Fludarabine (25 mg/m2 per day) and Cyclophosphamide (1000mg/m2 per day) on day -3. Doses of 9×10^9 cells, 13.5×10^9 anti-CD19 CAR NK cells (KN5501) will infused in each group using the dose-escalation strategy
| Study group | Role | What they receive |
|---|---|---|
| anti-CD19 CAR NK cells | Experimental | Not described Receives: Drug: anti-CD19 CAR NK cells (KN5501) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Dose Limiting Toxicity (DLTs)
To characterize the Dose Limiting Toxicities (DLTs) of anti-CD19 CAR NK Cells for refractory immune thrombocytopenia
Measured: within 4 weeks after infusion; 12, 24, 36 and 52 weeks after infusion
Treatment Emergent Adverse Events (TEAEs)
To characterize the Treatment Emergent Adverse Events (TEAEs) of anti-CD19 CAR NK Cells for refractory immune thrombocytopenia
Measured: within 4 weeks after infusion; 12, 24, 36 and 52 weeks after infusion
Secondary outcomes (1)
Objective Response Rate of subjects
Objective Response is assessed according to definitions of International ITP Working Group. Objective Response Rate is defined as proportion of patients with PLT content.
Measured: Time Frame: 2, 4, 8, 12, 16, 24, 28, 40 and 52 weeks after first infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2024
- Study start30 Aug 2024
- Primary completion30 Jul 2025 (estimated)
- Study completion30 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombocytopenia AlloimmuneStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Changzhou No.2 People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Rui Therapeutics Co., Ltd
- Responsible party
- Principal investigator, Lu Xuzhang, Changzhou No.2 People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06337474
- Sponsor's protocol ID
- [2023]YLJSA089
- Status last verified
- Apr 2025
- First submitted
- 19 Mar 2024
- Last updated
- 30 Apr 2025
- 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 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 Cell Injections for the Treatment of Refractory Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06337474
- Acronym / sponsor ID
- [2023]YLJSA089
- Conditions
- Thrombocytopenia Alloimmune
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Changzhou No.2 People's HospitalUniversity, hospital or other
- Collaborators
- Rui Therapeutics Co., Ltd
- Study start
- 30 Aug 2024 (actual)
- Primary completion
- 30 Jul 2025 (estimated)
- Study completion
- 30 Jul 2026 (estimated)
- First posted
- 29 Mar 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 arm, open-label pilot study is designed to determine the safety and effectiveness of CD19 CAR NK cells (KN5501) in patients with refractory immune thrombocytopenia. 9 patients are planned to be enrolled in the dose-escalation trial (9×10^9 cells, 13.5×10^9 cells). The primary objective of the study is to evaluation of the safety and feasibility of KN5501 for the treatment of relapsed/refractory B-cell related autoimmune diseases. The secondary objective is to evaluate evaluation of KN5501 for the treatment of refractory immune thrombocytopenia. The exploratory objective is to evaluate expansion, persistence and ability to deplete CD19 positive B cells of KN5501 in patients with refractory immune thrombocytopenia.
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 | Drug: anti-CD19 CAR NK cells (KN5501) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Age: ≥ 18 years old and ≤ 65 years old, male or female, subjects voluntarily participate in this clinical study and sign the Informed Consent Form (ICF);
- Diagnosis of immune thrombocytopenia (UTP) (according to American Society of Hematology 2019 guidelines and International ITP Working Group);
- Platelet count is decreased in routine blood tests at least two consecutive times, with no obvious abnormalities in the morphology of blood cells on microscopic examination of peripheral blood smears;
- Spleen is not enlarged during screening;
- The morphology of bone marrow cells in subjects is characterized by increased or normal megakaryocytes with impaired maturation;
Key exclusion criteria (13 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 active infection receiving intravenous (IV) antibiotic treatment, or received intravenous (IV) antibiotic treatment within one week prior to anti-CD19 CAR NK Cell (KN5501) infusion;
- Subjects with acquired and congenital immunodeficiency diseases;
- 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 cells (KN5501)Patients will receive Fludarabine (25 mg/m2 per day) and Cyclophosphamide (1000mg/m2 per day) on day -3. Doses of 9×10^9 cells, 13.5×10^9 anti-CD19 CAR NK cells (KN5501) will infused in each group using the dose-escalation strategy | Drug | anti-CD19 CAR NK cells |
Primary outcome
Incidence of Dose Limiting Toxicity (DLTs)
Time frame: within 4 weeks after infusion; 12, 24, 36 and 52 weeks after infusion
To characterize the Dose Limiting Toxicities (DLTs) of anti-CD19 CAR NK Cells for refractory immune thrombocytopenia
Treatment Emergent Adverse Events (TEAEs)
Time frame: within 4 weeks after infusion; 12, 24, 36 and 52 weeks after infusion
To characterize the Treatment Emergent Adverse Events (TEAEs) of anti-CD19 CAR NK Cells for refractory immune thrombocytopenia
1 secondary outcome
Objective Response Rate of subjects
Time frame: Time Frame: 2, 4, 8, 12, 16, 24, 28, 40 and 52 weeks after first 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 (KN5501) in people with Thrombocytopenia Alloimmune, run by Changzhou No.2 People's Hospital 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 12 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: 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 Xuzhang Lu, Doctor (+86-15295189493, Luxuzhang2008@163.com), 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 (DLTs) (time frame: within 4 weeks after infusion; 12, 24, 36 and 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.