Refractory Immune CytopeniaPhase 1NCT06787989
BCMA-CD19 CCAR T Cell Treatment of Refractory Immune Thrombocytopenia Associated with Autoimmune Diseases
A Phase 1 study testing BCMA-CD19 cCAR T cells in people with Refractory Immune Cytopenia, run by iCell Gene Therapeutics at 1 site in China.
Trial summary
This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of BCMA-CD19 cCAR T cells in patients with refractory ITP associated with autoimmune disease. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Refractory Immune Cytopenia
- What is being tested?
- BCMA-CD19 cCAR T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 31 Aug 2024to Apr 2027
- Phase
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 1. Age: 18~60 years old; 2. Diagnosed with immune thrombocytopenia associated with autoimmune diseases including systemic lupus erythematosus (according to the 1997 or 2009 ACR classification criteria), primary Sjogren's syndrome (according to the 2002 ACR/EULAR international classification criteria), undifferentiated connective tissue disease (according to the 1999 international classification criteria). Or patient without clinical manifestations related to connective tissue disease, but with positive anti nuclear antibodies (≥ 1:100) and/or without positive anti SSA/Ro-52 antibodies;Serum creatinine <221.0μmol/L (2.5mg/dl); 3. platelet count<30 × 10 ⁹/L or platelet count ≥ 20 × 10 ⁹/L, accompanied by bleeding symptoms (bleeding symptom score ≥ 2 points). No obvious active infection; 4. Voluntary participation and informed consent signed by the patient or his/her legal/authorized representative.
Who cannot join
- 1. Serious accompanying diseases that researchers consider clinically significant due to poor control, such as (but not limited to) neurological, cardiovascular, renal, liver, endocrine, or gastrointestinal diseases CNS disease: Active central nervous system (CNS) lupus (including epilepsy, psychosis, organic encephalopathy syndrome, cerebrovascular accident [CVA], encephalitis or CNS vasculitis), visual Disorders, cranial neuropathy requiring intervention 2. Abnormal liver function: aspartate transaminase (AST) or alanine transaminase (ALT) or glutamyl transpeptidase (GGT) detection value is greater than 1.5 times the upper limit of normal (ULN);; urinary protein quantification>1g/24h.
- 3. History of malignant tumors 4. Active hepatitis B or C.，and HIV positive 5. Individuals with a history of drug allergies or allergies. 6. Have any other clinically significant disease history or current disease that, in the judgment of the research physician, may pose a risk to the safety of the subjects, or interfere with the completion of the research procedure and the evaluation of safety and efficacy.
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
West China Hospital
Chengdu, Sichuan, 610041
Qibing Xie, MD, 011-86-18980601299, xieqibing1971@163.com
Min Yang, MD, 13618028207, yangmin7911@126.com
About this study
Official title: Treatment of Refractory Immune Thrombocytopenia
This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of BCMA-CD19 cCAR T cells in patients with refractory ITP associated with autoimmune disease.
Read the detailed description
Immune thrombocytopenia (ITP) Can be associated with various autoimmune diseases, including SLE, and SS. Patients with refractory thrombocytopenia often have long hospital stays, high medical costs, high demand for blood products, and are prone to complications of other systemic injuries. Such patients require active treatment to reduce the risk of life-threatening bleeding, delay the progression of the disease prognosis Glucocorticoids combined with immunosuppressive agents are still the main treatment strategies. Recently, biological agents targeting abnormal immune cells, such as rituximab and belimumab, which deplete B cells have also achieved some success in the treatment of ITP. However, these agents cannot permanently reverse the production of abnormal antibodies as they are unable to eliminate pathogenic long-lived plasma cells because these agents cannot penetrate lymph nodes and soft tissue. The BCMA-CD19 cCAR T-cells are designed to deplete antibody-producing 'root", B cells and plasma cells.
- Conditions
- Refractory Immune Cytopenia
- Being tested
- BCMA-CD19 cCAR T cells
- Keywords
- immune thrombocytopenia, CAR T cells, BCMA-CD19 cCAR, autoimmune diseaases
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCMA-CD19 cCAR T cellsBiological
- BCMA-CD19 cCAR T cells are used to treat patients. Patient will be administered either fresh or thawed CAR T cells by IV injection after receiving lymphodepleting chemotherapy.
| Study group | Role | What they receive |
|---|---|---|
| BCMA-CD19 cCAR T cells | Experimental | Dose escalation phase: patient's T cells will be transduced with a retroviral vector to express a BCMA-CD19 cCAR. with an escalation approach. Receives: Biological: BCMA-CD19 cCAR T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number and incidence of adverse events after BCMA-CD19 cCAR T cell infusion
Evaluation all possible adverse reactions, including the number, incidence, and severity of symptoms such as cytokine release syndromes and neurotoxicity within 3 months after BCMA-CD19 cCAR infusion.
Measured: 24 months
Secondary outcomes (1)
Overall remission rate 12 weeks after BCMA-CD19 cCAR
- Complete response (CR): Platelet count ≥ 100 × 109/L and no bleeding;
- Partial response (PR): platelet count<100 × 109/L, but increased by at least 2 times compared to baseline platelet count, with no bleeding symptoms
- Disease control: Disease control monitored up to 2 years after BCMA-CD19 cCAR T cells infusion)
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 posted22 Jan 2025
- Study start31 Aug 2024
- Primary completionAug 2026 (estimated)
- Study completionApr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Immune CytopeniaStudies 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iCell Gene Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- iCell ImmunityX（Hangzhou）Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06787989
- Sponsor's protocol ID
- ICG318-ITP-01
- Status last verified
- Jan 2025
- First submitted
- 8 Jan 2025
- Last updated
- 22 Jan 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 22 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Refractory Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06787989
- Acronym / sponsor ID
- ICG318-ITP-01
- Conditions
- Refractory Immune Cytopenia
- Keywords
- immune thrombocytopenia, CAR T cells, BCMA-CD19 cCAR, autoimmune diseaases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- iCell Gene TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- iCell ImmunityX（Hangzhou）Co., Ltd.
- Study start
- 31 Aug 2024 (actual)
- Primary completion
- Aug 2026 (estimated)
- Study completion
- Apr 2027 (estimated)
- First posted
- 22 Jan 2025 (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 phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of BCMA-CD19 cCAR T cells in patients with refractory ITP associated with autoimmune disease.
Detailed description
Immune thrombocytopenia (ITP) Can be associated with various autoimmune diseases, including SLE, and SS. Patients with refractory thrombocytopenia often have long hospital stays, high medical costs, high demand for blood products, and are prone to complications of other systemic injuries. Such patients require active treatment to reduce the risk of life-threatening bleeding, delay the progression of the disease prognosis Glucocorticoids combined with immunosuppressive agents are still the main treatment strategies. Recently, biological agents targeting abnormal immune cells, such as rituximab and belimumab, which deplete B cells have also achieved some success in the treatment of ITP. However, these agents cannot permanently reverse the production of abnormal antibodies as they are unable to eliminate pathogenic long-lived plasma cells because these agents cannot penetrate lymph nodes and soft tissue. The BCMA-CD19 cCAR T-cells are designed to deplete antibody-producing 'root", B cells and plasma cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BCMA-CD19 cCAR T cells | Experimental | Biological: BCMA-CD19 cCAR T cellsDose escalation phase: patient's T cells will be transduced with a retroviral vector to express a BCMA-CD19 cCAR. with an escalation approach. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- 1. Age: 18~60 years old; 2. Diagnosed with immune thrombocytopenia associated with autoimmune diseases including systemic lupus erythematosus (according to the 1997 or 2009 ACR classification criteria), primary Sjogren's syndrome (according to the 2002 ACR/EULAR international classification criteria), undifferentiated connective tissue disease (according to the 1999 international classification criteria). Or patient without clinical manifestations related to connective tissue disease, but with positive anti nuclear antibodies (≥ 1:100) and/or without positive anti SSA/Ro-52 antibodies;Serum creatinine <221.0μmol/L (2.5mg/dl); 3. platelet count<30 × 10 ⁹/L or platelet count ≥ 20 × 10 ⁹/L, accompanied by bleeding symptoms (bleeding symptom score ≥ 2 points). No obvious active infection; 4. Voluntary participation and informed consent signed by the patient or his/her legal/authorized representative.
Key exclusion criteria (2 listed)
- 1. Serious accompanying diseases that researchers consider clinically significant due to poor control, such as (but not limited to) neurological, cardiovascular, renal, liver, endocrine, or gastrointestinal diseases CNS disease: Active central nervous system (CNS) lupus (including epilepsy, psychosis, organic encephalopathy syndrome, cerebrovascular accident [CVA], encephalitis or CNS vasculitis), visual Disorders, cranial neuropathy requiring intervention 2. Abnormal liver function: aspartate transaminase (AST) or alanine transaminase (ALT) or glutamyl transpeptidase (GGT) detection value is greater than 1.5 times the upper limit of normal (ULN);; urinary protein quantification>1g/24h.
- 3. History of malignant tumors 4. Active hepatitis B or C.，and HIV positive 5. Individuals with a history of drug allergies or allergies. 6. Have any other clinically significant disease history or current disease that, in the judgment of the research physician, may pose a risk to the safety of the subjects, or interfere with the completion of the research procedure and the evaluation of safety and efficacy.
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 |
|---|---|---|
| BCMA-CD19 cCAR T cells• BCMA-CD19 cCAR T cells are used to treat patients. Patient will be administered either fresh or thawed CAR T cells by IV injection after receiving lymphodepleting chemotherapy. | Biological | BCMA-CD19 cCAR T cells |
Primary outcome
The number and incidence of adverse events after BCMA-CD19 cCAR T cell infusion
Time frame: 24 months
Evaluation all possible adverse reactions, including the number, incidence, and severity of symptoms such as cytokine release syndromes and neurotoxicity within 3 months after BCMA-CD19 cCAR infusion.
1 secondary outcome
Overall remission rate 12 weeks after BCMA-CD19 cCAR
Time frame: 24 months
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 22 Jan 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 Phase 1 study testing BCMA-CD19 cCAR T cells in people with Refractory Immune Cytopenia, run by iCell Gene Therapeutics 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 1 inclusion and 2 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 22 Jan 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?
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.
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 Qibing Xie, MD (0118618980601299, xieqibing1971@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number and incidence of adverse events after BCMA-CD19 cCAR T cell infusion (time frame: 24 months). 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 22 Jan 2025.