SclerodermaEarly Phase 1NCT05085444
A Study of CD19/BCMA Chimeric Antigen Receptor T Cells Therapy for Patients With Refractory Scleroderma
A Early Phase 1 study testing Assigned Interventions CD19/BCMA CAR T-cells in people with Scleroderma and Autoimmune Diseases, run by Zhejiang University at 1 site in China.
Trial summary
A Study of CD19/BCMA Chimeric Antigen Receptor T Cells Therapy for Patients With Refractory Scleroderma Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Scleroderma, Autoimmune Diseases
- What is being tested?
- Assigned Interventions CD19/BCMA CAR T-cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 8 Oct 2021to 8 Oct 2024
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Scleroderma with positive CD19/BCMA expression , and the conventional treatment is not effective and (or) no effective treatment
- Estimated survival time> 12 weeks;
- Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up;
- Patients or their legal guardians volunteer to participate in the study and sign the informed consent.
Who cannot join
- Subjects with any of the following exclusion criteria were not eligible for this trial:
- History of craniocerebral trauma, conscious disturbance, epilepsy, cerebrovascular ischemia, and cerebrovascular, hemorrhagic diseases;
- Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythmia in the past;
- Pregnant (or lactating) women;
- Patients with severe active infections (excluding simple urinary tract infection and bacterial pharyngitis);
- Active infection of hepatitis B virus or hepatitis C virus;
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- Concurrent therapy with systemic steroids within 2 weeks prior to screening, except for the patients recently or currently receiving in haled steroids;
- Creatinine>2.5mg/dl, or ALT / AST > 3 times of normal amounts, or bilirubin>2.0 mg/dl;
- Other uncontrolled diseases that were not suitable for this trial;
- Patients with HIV infection;
- Any situations that the investigator believes may increase the risk of patients or interfere with the results of study
- Platelets ≥30×10E9/L, and absolute lymphocyte count ≥1.0×10E9/L
- Methylprednisolone (maximum dose 1mg/kg) or prednisone (maximum dose 1.25mg/kg) instead of immunosuppressive agents to control the disease.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital, College of Medicine, Zhejiang University
Hangzhou, Zhejiang, 310003
He Huang, PhD, 86-13605714822, hehuangyu@126.com
Yongxian Hu, PhD, 86-15957162012, huyongxian2000@aliyun.com
About this study
A Study of CD19/BCMA Chimeric Antigen Receptor T Cells Therapy for Patients With Refractory Scleroderma
Read the detailed description
Autoimmune diseases only show local pathological damage, but more often systemic lesions. If not diagnosed and treated in time or poorly controlled, a risk of disability or even death as the course of the disease progresses. Studies have shown that B cells can present their own antigens to autoimmune T cells to promote the release of inflammatory factors, or they can differentiate into plasma cells to release autoantibodies, and play an important role in the occurrence and progression of autoimmune diseases. In recent years, it has become a major research focus to deplete B cells in patients or inhibit B cell function. This research focuses on CAR-T cells killing B cells. This fully reflects the application prospects of CAR-T cells in autoimmune diseases.
Based on the current research progress, our center intends to conduct research on the safety and effectiveness of CD19/BCMA CAR-T cells in the treatment of refractory scleroderma
- Conditions
- Scleroderma, Autoimmune Diseases
- Being tested
- Assigned Interventions CD19/BCMA CAR T-cells
- Keywords
- Scleroderma, CD19 CAR T-cell therapy, BCMA CAR T-cell therapy
- Medical subject headings
- Scleroderma, Diffuse, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Assigned Interventions CD19/BCMA CAR T-cellsBiological
Drug: CD19/BCMA CAR T-cells Each subject receive CD19/BCMA CAR T-cells by intravenous infusion Other Name: CD19/BCMA CAR T-cells injection
| Study group | Role | What they receive |
|---|---|---|
| Treatment of Scleroderma | Experimental | Experimental：Administration of CD19/BCMA CAR T-cells A dose levels of 1-4*10E6/kg are administrated for each subject. Receives: Biological: Assigned Interventions CD19/BCMA CAR T-cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-limiting toxicity (DLT)
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Measured: Baseline up to 28 days after CD19/BCMA CAR T-cells infusion
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events [Safety and Tolerability]
Measured: Up to 90 days after CD19/BCMA CAR T-cells infusion
Secondary outcomes (6)
Concentration of CAR-T cells
In peripheral blood
Measured: From admission to the end of the follow-up, up to 2 years
Objective Response Rate, ORR
Proportion of subjects with complete or partial remission
Measured: In 3 months of CD19/BCMA CAR-T cell infusion
Disease control rate, DCR
The percentage of patients with remission and stable disease after treatment in the total evaluable cases.
Measured: From Day 28 CD19/BCMA CAR-T infusion up to 2 years
Duration of remission, DOR
The time from the first assessment of remission or partial remission of the disease to the first assessment of disease progression or death from any cause
Measured: 24 months post CD19/BCMA CAR-T cells infusion
Progression-free survival, PFS
The time from cell reinfusion to the first assessment of disease progression or death from any cause
Measured: 24 months post CD19/BCMA CAR-Tcells infusion
Overall survival, OS
The time from the cell reinfusion to death due to any cause
Measured: From CD19/BCMA CAR-T infusion to death，up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2021
- Study start8 Oct 2021
- Primary completion8 Oct 2024 (estimated)
- Study completion8 Oct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SclerodermaStudies of the same condition, open and under way.
- More trials run by Zhejiang 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
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Responsible party
- Principal investigator, He Huang, Zhejiang University
- Investigators
- He Huang, PhD, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05085444
- Sponsor's protocol ID
- CD19/BCMA-002
- Status last verified
- Oct 2021
- First submitted
- 11 Oct 2021
- Last updated
- 20 Oct 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of CD19/BCMA Chimeric Antigen Receptor T Cells Therapy for Patients With Refractory Scleroderma
- ClinicalTrials.gov ID
- NCT05085444
- Acronym / sponsor ID
- CD19/BCMA-002
- Conditions
- Scleroderma, Autoimmune Diseases
- Keywords
- Scleroderma, CD19 CAR T-cell therapy, BCMA CAR T-cell therapy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Study start
- 8 Oct 2021 (actual)
- Primary completion
- 8 Oct 2024 (estimated)
- Study completion
- 8 Oct 2024 (estimated)
- First posted
- 20 Oct 2021 (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 Study of CD19/BCMA Chimeric Antigen Receptor T Cells Therapy for Patients With Refractory Scleroderma
Detailed description
Autoimmune diseases only show local pathological damage, but more often systemic lesions. If not diagnosed and treated in time or poorly controlled, a risk of disability or even death as the course of the disease progresses. Studies have shown that B cells can present their own antigens to autoimmune T cells to promote the release of inflammatory factors, or they can differentiate into plasma cells to release autoantibodies, and play an important role in the occurrence and progression of autoimmune diseases. In recent years, it has become a major research focus to deplete B cells in patients or inhibit B cell function. This research focuses on CAR-T cells killing B cells. This fully reflects the application prospects of CAR-T cells in autoimmune diseases.
Based on the current research progress, our center intends to conduct research on the safety and effectiveness of CD19/BCMA CAR-T cells in the treatment of refractory scleroderma
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment of Scleroderma | Experimental | Biological: Assigned Interventions CD19/BCMA CAR T-cellsExperimental：Administration of CD19/BCMA CAR T-cells A dose levels of 1-4*10E6/kg are administrated for each subject. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Scleroderma with positive CD19/BCMA expression , and the conventional treatment is not effective and (or) no effective treatment
- Estimated survival time> 12 weeks;
- Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up;
- Patients or their legal guardians volunteer to participate in the study and sign the informed consent.
Key exclusion criteria (13 listed)
- Subjects with any of the following exclusion criteria were not eligible for this 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 |
|---|---|---|
| Assigned Interventions CD19/BCMA CAR T-cellsDrug: CD19/BCMA CAR T-cells Each subject receive CD19/BCMA CAR T-cells by intravenous infusion Other Name: CD19/BCMA CAR T-cells injection | Biological | Treatment of Scleroderma |
Primary outcome
Dose-limiting toxicity (DLT)
Time frame: Baseline up to 28 days after CD19/BCMA CAR T-cells infusion
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Up to 90 days after CD19/BCMA CAR T-cells infusion
Incidence of treatment-emergent adverse events [Safety and Tolerability]
6 secondary outcomes
Concentration of CAR-T cells
Time frame: From admission to the end of the follow-up, up to 2 years
Objective Response Rate, ORR
Time frame: In 3 months of CD19/BCMA CAR-T cell infusion
Disease control rate, DCR
Time frame: From Day 28 CD19/BCMA CAR-T infusion up to 2 years
Duration of remission, DOR
Time frame: 24 months post CD19/BCMA CAR-T cells infusion
Progression-free survival, PFS
Time frame: 24 months post CD19/BCMA CAR-Tcells infusion
Overall survival, OS
Time frame: From CD19/BCMA CAR-T infusion to death，up to 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2021.
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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.
He Huang, PhD principal investigator
First Affiliated Hospital of Zhejiang University
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 Assigned Interventions CD19/BCMA CAR T-cells in people with Scleroderma and Autoimmune Diseases, run by Zhejiang 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 4 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: status not confirmed, as of the sponsor's last update on 20 Oct 2021. 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 He Huang, PhD (86-13605714822, hehuangyu@126.com), 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: Baseline up to 28 days after CD19/BCMA CAR T-cells 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 20 Oct 2021.