Systemic Sclerosis (SSc)Phase 1NCT06792344
Clinical Study of CD19 CAR-T in the Treatment of Refractory Systemic Sclerosis
A Phase 1 study testing anti-CD19 CAR-T cells in people with Systemic Sclerosis (SSc), run by The Children's Hospital of Zhejiang University School of Medicine at 2 sites in 2 countries.
Trial summary
This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of childhood-onset systemic sclerosis Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Systemic Sclerosis (SSc)
- What is being tested?
- anti-CD19 CAR-T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 14 Dec 2025to 31 Dec 2028
- Phase
- 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 5 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Gender unlimited, age ≥5 years;
- Meet the classification criteria for Systemic sclerosis (SSc) as defined by the 2013ACR/EULAR standards;
- any one of the anti-nuclear antibodies or systemic sclerosis specific antibodies positive;
- Improved Rodnan skin score (mRSS) ≥15 points (total 51 points);
- Meet the definition of intractable disease: Glucocorticoids (≥0.5mg/kg/d) and cyclophosphamide, as well as one or more of the following immunomodulators (including antimalarial drugs, azathioprine,motecophanate, methotrexate, leflunomide, tachlimus, cyclosporine, and biologics including rituximab, Beliumab, and titacept, etc.), did not show significant remission of the disease for more than 3 months; Or meet the criteria for rapid disease progression , clinical routine treatment is ineffective, and the benefits outweigh the risks as determined by the investigator and the patient's or guardian's full and informed consent can be considered for inclusion.
- Major organ functions were basically normal: left ventricular ejection fraction (LVEF) ≥55%, no obvious abnormality in electrocardiogram; eGFR≥30ML/min/1.73m2；AST and ALT≤3.0ULN, total bilirubin ≤2.0×ULN; SpO2≥92%, DLCO≥ 40% of predicted value, FVC≥ 50% of predicted value;
- The subject of childbearing age has a negative urine pregnancy test result and agrees to take effective contraceptive measures during the test period until 1 year after the infusion;
- The patient or his/her guardian agrees to participate in the clinical trial and signs an informed consent indicating that he/she understands the purpose and procedure of the clinical trial and is willing to participate in the study
Who cannot join
- （1） Severe pulmonary hypertension (PHA) (mean pulmonary pressure > 45mmHg) or other severe major organ involvement; (2) have previously received CAR T cell therapy (except those whose safety risks have been ruled out by the investigators); (3) patients with active central nervous system diseases, such as epilepsy, cerebrovascular ischemia/hemorrhage, dementia, cerebellar diseases, etc.; (4) Congenital heart disease or history of acute myocardial infarction within 6 months before screening, or severe arrhythmias (including multi-source frequent supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with a large number of pericardial effusion, serious myocarditis, etc.; Or patients with unstable vital signs who require vasoactive drugs to maintain blood pressure; (5) Active tuberculosis at the time of screening; (6) There are active infections or uncontrollable infections that require systemic treatment at the time of screening; (7) Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; Acute graft-versus-host disease (GVHD) of grade 2 or above was present within 2 weeks prior to screening; (8) Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA detection greater than the normal range; Hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Positive for human immunodeficiency virus (HIV) antibodies; Treponema pallidum antibody positive; (9) Had received live vaccine within 4 weeks before screening; (10) Positive blood pregnancy test; (11) Patients with known malignant diseases such as tumors before screening; (12) Patients who had participated in other interventional clinical trials within 3 months before enrollment; (13) Situations in which other investigators consider it inappropriate to participate in the study
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
China1 site
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 0571
Christmas Island1 site
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310053
About this study
Official title: Clinical Study of CD19 Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Refractory Systemic Sclerosis
This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of childhood-onset systemic sclerosis
Read the detailed description
Systemic sclerosis (SSc) is a type of systemic autoimmune disease characterized by skin thickening and hardening. In addition to skin involvement, it can also affect the internal organs (lungs, cardiovascular, kidney, digestive tract, etc.).
Currently there are no drugs that can affect the natural course of systemic sclerosis, and individualized treatment according to target organ involvement is usually used, glucocorticoid is used for patient combined with myositis or mixed connective tissue , and various immunosuppressants, including azathioprine, mycophenolate and cyclophosphamide, may be helpful in the treatment of interstitial lung disease. The anti-IL-6 blocker tocilizumab and the anti-fibrotic drug nitidanib have also been shown to be effective to SSC-associated interstitiallungdisease (ILD) and cardiac involvment.
Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Clinical studies have demonstrated that targeted CD19 CAR-T cells hold significant therapeutic potential for SLE. These cells effectively slow down the pathological progression of SLE and can also effectively treat severe cases. Furthermore, targeted CD19 CAR-T cells are also expected to restore the immune system in SLE patients, potentially allowing them to discontinue lifelong medication and avoid serious long-term side effects of drugs like hormones and immunosuppressants. Studies have reported that CAR-T has a good therapeutic effect on a variety of autoimmune diseases such as systemic sclerosis and idiopathic inflammatory dermatomyositis.The purpose of this study is to assess the safety and efficacy of the anti-CD19 CAR-T cells in the treatment of childhood-onset refractory SSc.
- Conditions
- Systemic Sclerosis (SSc)
- Being tested
- anti-CD19 CAR-T cells
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-CD19 CAR-T cellsOther
Intravenous injection the targeting CD19 CAR T cell
anti-CD19 CAR-T cellsDrug
CD19-targeting CAR-T cells
| Study group | Role | What they receive |
|---|---|---|
| anti-CD19 CAR-T cells in the treatment of SSc | Experimental | Not described Receives: Other: anti-CD19 CAR-T cells; Drug: anti-CD19 CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy endpoint
the Change in the modified Rodnan Skin Score (mRSS) from baseline at 6 months
Measured: 6 months
Secondary outcomes (2)
efficiency endpoint
The EUSTAR activity index and combined response index (CRISS) changed after 1 month ,2 months ,6 months,12 months
Measured: the first month ,the second month ,the sixth month
PK parameter
The maximum concentration (Cmax) of CD19-targeted CAR T cells in blood after infusion, the time to reach the maximum concentration (Tmax) and the area under the curve at 28 days /90 days after infusion were AUC28d/90d
Measured: 28 days and 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2025
- Study start14 Dec 2025
- Primary completion14 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Why it stopped: temporarily suspended due to no new subjects at present
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Sclerosis (SSc)Studies of the same condition, open and under way.
- More trials run by The Children's Hospital of Zhejiang University School of MedicineThe 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- CD19 CAR-T
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Guangzhou Women and Children's Medical Center
- Responsible party
- Principal investigator, Mao Jianhua, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06792344
- Sponsor's protocol ID
- 2024-IRB-0306-P-01
- Status last verified
- Dec 2025
- First submitted
- 23 Dec 2024
- Last updated
- 8 Sep 2026
- 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 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of CD19 Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Refractory Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT06792344
- Acronym / sponsor ID
- 2024-IRB-0306-P-01
- Conditions
- Systemic Sclerosis (SSc)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: temporarily suspended due to no new subjects at present
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- The Children's Hospital of Zhejiang University School of MedicineUniversity, hospital or other
- Collaborators
- Guangzhou Women and Children's Medical Center
- Study start
- 14 Dec 2025 (actual)
- Primary completion
- 14 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 24 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 an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of childhood-onset systemic sclerosis
Detailed description
Systemic sclerosis (SSc) is a type of systemic autoimmune disease characterized by skin thickening and hardening. In addition to skin involvement, it can also affect the internal organs (lungs, cardiovascular, kidney, digestive tract, etc.).
Currently there are no drugs that can affect the natural course of systemic sclerosis, and individualized treatment according to target organ involvement is usually used, glucocorticoid is used for patient combined with myositis or mixed connective tissue , and various immunosuppressants, including azathioprine, mycophenolate and cyclophosphamide, may be helpful in the treatment of interstitial lung disease. The anti-IL-6 blocker tocilizumab and the anti-fibrotic drug nitidanib have also been shown to be effective to SSC-associated interstitiallungdisease (ILD) and cardiac involvment.
Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Clinical studies have demonstrated that targeted CD19 CAR-T cells hold significant therapeutic potential for SLE. These cells effectively slow down the pathological progression of SLE and can also effectively treat severe cases. Furthermore, targeted CD19 CAR-T cells are also expected to restore the immune system in SLE patients, potentially allowing them to discontinue lifelong medication and avoid serious long-term side effects of drugs like hormones and immunosuppressants. Studies have reported that CAR-T has a good therapeutic effect on a variety of autoimmune diseases such as systemic sclerosis and idiopathic inflammatory dermatomyositis.The purpose of this study is to assess the safety and efficacy of the anti-CD19 CAR-T cells in the treatment of childhood-onset refractory SSc.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- CD19 CAR-T
| Arm | Type | Receives |
|---|---|---|
| anti-CD19 CAR-T cells in the treatment of SSc | Experimental | Other: anti-CD19 CAR-T cells, Drug: anti-CD19 CAR-T cells |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Gender unlimited, age ≥5 years;
- Meet the classification criteria for Systemic sclerosis (SSc) as defined by the 2013ACR/EULAR standards;
- any one of the anti-nuclear antibodies or systemic sclerosis specific antibodies positive;
- Improved Rodnan skin score (mRSS) ≥15 points (total 51 points);
- Meet the definition of intractable disease: Glucocorticoids (≥0.5mg/kg/d) and cyclophosphamide, as well as one or more of the following immunomodulators (including antimalarial drugs, azathioprine,motecophanate, methotrexate, leflunomide, tachlimus, cyclosporine, and biologics including rituximab, Beliumab, and titacept, etc.), did not show significant remission of the disease for more than 3 months; Or meet the criteria for rapid disease progression , clinical routine treatment is ineffective, and the benefits outweigh the risks as determined by the investigator and the patient's or guardian's full and informed consent can be considered for inclusion.
Key exclusion criteria (1 listed)
- （1） Severe pulmonary hypertension (PHA) (mean pulmonary pressure > 45mmHg) or other severe major organ involvement; (2) have previously received CAR T cell therapy (except those whose safety risks have been ruled out by the investigators); (3) patients with active central nervous system diseases, such as epilepsy, cerebrovascular ischemia/hemorrhage, dementia, cerebellar diseases, etc.; (4) Congenital heart disease or history of acute myocardial infarction within 6 months before screening, or severe arrhythmias (including multi-source frequent supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with a large number of pericardial effusion, serious myocarditis, etc.; Or patients with unstable vital signs who require vasoactive drugs to maintain blood pressure; (5) Active tuberculosis at the time of screening; (6) There are active infections or uncontrollable infections that require systemic treatment at the time of screening; (7) Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; Acute graft-versus-host disease (GVHD) of grade 2 or above was present within 2 weeks prior to screening; (8) Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA detection greater than the normal range; Hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Positive for human immunodeficiency virus (HIV) antibodies; Treponema pallidum antibody positive; (9) Had received live vaccine within 4 weeks before screening; (10) Positive blood pregnancy test; (11) Patients with known malignant diseases such as tumors before screening; (12) Patients who had participated in other interventional clinical trials within 3 months before enrollment; (13) Situations in which other investigators consider it inappropriate to participate in the study
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-T cellsIntravenous injection the targeting CD19 CAR T cell | Other | anti-CD19 CAR-T cells in the treatment of SSc |
| anti-CD19 CAR-T cellsCD19-targeting CAR-T cells | Drug | anti-CD19 CAR-T cells in the treatment of SSc |
Primary outcome
Efficacy endpoint
Time frame: 6 months
the Change in the modified Rodnan Skin Score (mRSS) from baseline at 6 months
2 secondary outcomes
efficiency endpoint
Time frame: the first month ,the second month ,the sixth month
PK parameter
Time frame: 28 days and 90 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Systemic Sclerosis (SSc)
RecruitingObservational study
Quantification of Calcinosis Cutis Disease Burden Using Computed Tomography Images
Systemic Sclerosis (SSc), Calcinosis Cutis
RecruitingPhase 1/2
Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders
Autoimmune Disorders, Systemic Sclerosis
RecruitingPhase 1
A Study to Investigate the Safety and Preliminary Efficacy of ALLO-329, an Allogeneic CAR T-cell Therapy, in Adults With Autoimmune Disease
Systemic Lupus Erythematosus (With and Without Nephritis), Idiopathic Inflammatory Myopathy
Not yet recruitingPhase 1
A Phase 1 Study of QLS2416 Injection in Participants With Autoimmune Diseases
Systemic Lupus Erythematosus, Rheumatoid Arthritis (RA)
Recruiting
Tissue Modeling in Systemic Sclerosis Using Induced Pluripotent Stem Cells (iPSCs)
Systemic Sclerosis (SSc)
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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing anti-CD19 CAR-T cells in people with Systemic Sclerosis (SSc), run by The Children's Hospital of Zhejiang University School of Medicine at 2 sites in 2 countries.
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 8 inclusion and 1 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: paused, as of the sponsor's last update on 8 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 2 sites in China, Christmas Island. 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?
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?
Efficacy endpoint (time frame: 6 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 8 Sep 2026.