Dermatomyositis, JuvenilePhase 1NCT06686524
Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
A Phase 1 study testing Anti-CD19 UCAR-T cells in people with Dermatomyositis, Juvenile and Dermatomyositis, run by Chongqing Precision Biotech Co., Ltd at 1 site in China.
Trial summary
This study is an open-label, single-arm, dose-escalation trial primarily designed to evaluate the safety and efficacy of a universal CAR-T cell therapy targeting CD19 in the treatment of patients with RJDM. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Dermatomyositis, Juvenile, Dermatomyositis
- What is being tested?
- Anti-CD19 UCAR-T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 11 Nov 2024to 10 Nov 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- (1) Age ≥ 5 years old;
- (2) Diagnosis of juvenile dermatomyositis (JDM) according to Bohan and Peter criteria;
- (3) Meeting the classification criteria for RJDM, and meeting condition ① and any one of ② to ⑤ conditions:
- Intolerance or inadequate response to glucocorticoids (prednisone 1-2mg/kg/d or equivalent dose of other hormones) and at least two immunosuppressants, with hormone therapy lasting for at least 6 months;
- Rapid progression of the disease and/or involvement of organs such as lungs, heart, and gastrointestinal tract;
- Calcification of subcutaneous or muscular and articular tissues;
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- Repeated skin rashes or ulcers;
- Repeated or persistent muscle weakness (muscle magnetic resonance imaging indicating widespread, diffuse edema or a Children's Myositis Assessment Scale (CMAS) score < 48, and at least two abnormal results among the following five core measurement indicators: Physician's Global Assessment (PhGA) ≥ 2cm, Patient's Global Assessment (PtGA) ≥ 2cm, Disease Activity Score (DAS) ≥ 2 points, Child Health Assessment Questionnaire (C-HAQ) total score ≥ 0.25 points, and muscle enzyme level > 1.5 times the upper limit of normal (ULN));
- (4)Patients with immune-mediated necrotizing myopathy who are positive for SRP or HMGCR antibodies meet the criteria for RJDM and can be directly included;
- (5) Basic normal function of important organs:
- Cardiac function: Left ventricular ejection fraction (LVEF) ≥55%, with no significant abnormalities observed in the electrocardiogram;
- Renal function: eGFR ≥ 30mL/min/1.73m2;
- Liver function: AST and ALT ≤3.0 ULN, total bilirubin ≤2.0×ULN (excluding those caused by primary diseases);
- Pulmonary function: SpO2 ≥92%;
- (6) Female subjects of childbearing age have a negative result in the urine pregnancy test and agree to take effective contraceptive measures during the trial until one year after infusion;
- (7) The patient or their guardian agrees to participate in this clinical trial and signs an informed consent form, indicating their understanding of the purpose and procedures of the clinical trial and their willingness to participate in the study.
Who cannot join
- Subjects meeting any of the following criteria will be excluded from this trial:
- (1) Previously received CAR-T cell therapy (except for those whose safety risks have been judged as eliminated by the investigator);
- (2) Accompanied by primary immunodeficiency disease or severe secondary immunodeficiency disease that has not been corrected;
- (3) Accompanied by severe, active, or uncontrolled infectious diseases, including but not limited to active tuberculosis, latent tuberculous infection, active viral hepatitis, etc.;
- (4) Known to have active malignant diseases or confirmed malignancies before screening (including hematological malignancies and solid tumors, except for resected and cured cutaneous basal cell carcinoma);
- (5) Suffering from congenital heart disease or having a history of acute myocardial infarction within 6 months, or severe arrhythmia (including multifocal frequent ventricular supraventricular tachycardia, ventricular tachycardia, etc.); or complicated with moderate to large pericardial effusion, severe myocarditis, etc.; or unstable vital signs requiring vasopressors to maintain blood pressure;
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- (6) Accompanied by other diseases that require long-term use of glucocorticoids or immunosuppressants;
- (7) Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; or presence of acute graft-versus-host disease (GVHD) of grade 2 or above within 2 weeks before screening;
- (8) Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and peripheral blood hepatitis B virus (HBV) DNA titer detection is greater than the normal reference range; or positive for hepatitis C virus (HCV) antibody and peripheral blood hepatitis C virus (HCV) RNA titer detection is greater than the normal reference range; or positive for human immunodeficiency virus (HIV) antibody; or positive for syphilis test;
- (9) Received live vaccines within 4 weeks before screening;
- (10) Positive blood pregnancy test;
- (11) Participated in other clinical trials within 3 months before enrollment;
- (12) Other conditions that the investigator considers unsuitable for participation in the study.
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
Children's Hospital Zhejiang University School of Medicine, Hangzhou, zhejiang
Hangzhou, Zhejiang
Meiping Lu, M.D, +86 13685773988, meipinglu@zju.edu.cn
About this study
This study is an open-label, single-arm, dose-escalation trial primarily designed to evaluate the safety and efficacy of a universal CAR-T cell therapy targeting CD19 in the treatment of patients with RJDM. Additionally, the study aims to characterize its pharmacokinetic and pharmacodynamic properties, explore its role in immune system reconstitution, and assess long-term survival benefits.
Read the detailed description
This study adopts the "3+3" design, with a total of three dosage groups (1 × 107/kg, 3 × 107/kg, 6 × 107/kg). The study will start from the low dose group. Only when the present dose group has completed enrollment and no DLT has been observed, the study could escalate to the next dose group based on the evaluation of efficacy and cell kinetic data. If no DLT is observed even in the highest dose group, the escalation will be terminated.Each dose group is expected to enroll 3 to 6 patients, with a total of 9 to 18 patients anticipated in the trial.
- Conditions
- Dermatomyositis, Juvenile, Dermatomyositis
- Being tested
- Anti-CD19 UCAR-T cells
- Keywords
- Dermatomyositis, CD19, UCAR-T, Universal CAR-T
- Medical subject headings
- Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-CD19 UCAR-T cellsDrug
A single infusion of CD19 UCAR-T cells will be administered intravenously after lymphodepletion chemotherapy.
| Study group | Role | What they receive |
|---|---|---|
| Anti-CD19 UCAR-T cells | Experimental | Not described Receives: Drug: Anti-CD19 UCAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
The incidence of adverse events after CAR-T cell infusion was assessed by CTCAE, version 5.0.
Measured: 28 dyas
Total improvement score (TIS) after anti-CD19 UCAR-T cells infusion.
The total improvement score between 0-100 also corresponds to the degree of improvement, with higher improvement scores indicating greater improvement.
Measured: 6 months
Disease Activity Score (DAS) after anti-CD19 UCAR-T cells infusion.
Including musculoskeletal system score and skin mucosal score, with a total score of 20 points. The higher the score, the higher the disease activity
Measured: 6 months
Changes of myositis specific antibody levels and serum muscle enzyme levels after anti-CD19 UCAR-T cells infusion.
Measured: 6 months
Improvement in muscle inflammation infiltration by MRI after anti-CD19 UCAR-T cells infusion.
Measured: 6 months
Childhood Myositis Assessment Scale (CMAS) score after anti-CD19 UCAR-T cells
The total score is 52 points, the lower the score, the weaker the muscle strength and the more severe the disease.
Measured: 6 months
Secondary outcomes (4)
Cmax of anti-CD19 UCAR-T cells [Cell dynamics]
Measured: 1 month
Tmax of anti-CD19 UCAR-T cells [Cell dynamics]
Measured: 1 month
AUCs of anti-CD19 UCAR-T cells [Cell dynamics]
Measured: 1 month
Changes of myth enzyme after anti-CD19 UCAR-T cells
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2024
- Study start11 Nov 2024 (estimated)
- Primary completion10 Nov 2026 (estimated)
- Study completion10 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dermatomyositis, JuvenileStudies of the same condition, open and under way.
- More trials run by Chongqing Precision Biotech Co., LtdThe 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
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chongqing Precision Biotech Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06686524
- Sponsor's protocol ID
- PBC081
- Status last verified
- Nov 2024
- First submitted
- 11 Nov 2024
- Last updated
- 13 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
- ClinicalTrials.gov ID
- NCT06686524
- Acronym / sponsor ID
- PBC081
- Conditions
- Dermatomyositis, Juvenile, Dermatomyositis
- Keywords
- Dermatomyositis, CD19, UCAR-T, Universal CAR-T
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Chongqing Precision Biotech Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 11 Nov 2024 (estimated)
- Primary completion
- 10 Nov 2026 (estimated)
- Study completion
- 10 Nov 2027 (estimated)
- First posted
- 13 Nov 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.
This study is an open-label, single-arm, dose-escalation trial primarily designed to evaluate the safety and efficacy of a universal CAR-T cell therapy targeting CD19 in the treatment of patients with RJDM. Additionally, the study aims to characterize its pharmacokinetic and pharmacodynamic properties, explore its role in immune system reconstitution, and assess long-term survival benefits.
Detailed description
This study adopts the "3+3" design, with a total of three dosage groups (1 × 107/kg, 3 × 107/kg, 6 × 107/kg). The study will start from the low dose group. Only when the present dose group has completed enrollment and no DLT has been observed, the study could escalate to the next dose group based on the evaluation of efficacy and cell kinetic data. If no DLT is observed even in the highest dose group, the escalation will be terminated.Each dose group is expected to enroll 3 to 6 patients, with a total of 9 to 18 patients anticipated in the trial.
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 UCAR-T cells | Experimental | Drug: Anti-CD19 UCAR-T cells |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- (1) Age ≥ 5 years old;
- (2) Diagnosis of juvenile dermatomyositis (JDM) according to Bohan and Peter criteria;
- (3) Meeting the classification criteria for RJDM, and meeting condition ① and any one of ② to ⑤ conditions:
- (4)Patients with immune-mediated necrotizing myopathy who are positive for SRP or HMGCR antibodies meet the criteria for RJDM and can be directly included;
- (5) Basic normal function of important organs:
Key exclusion criteria (13 listed)
- (1) Previously received CAR-T cell therapy (except for those whose safety risks have been judged as eliminated by the investigator);
- (2) Accompanied by primary immunodeficiency disease or severe secondary immunodeficiency disease that has not been corrected;
- (3) Accompanied by severe, active, or uncontrolled infectious diseases, including but not limited to active tuberculosis, latent tuberculous infection, active viral hepatitis, etc.;
- (4) Known to have active malignant diseases or confirmed malignancies before screening (including hematological malignancies and solid tumors, except for resected and cured cutaneous basal cell carcinoma);
- (5) Suffering from congenital heart disease or having a history of acute myocardial infarction within 6 months, or severe arrhythmia (including multifocal frequent ventricular supraventricular tachycardia, ventricular tachycardia, etc.); or complicated with moderate to large pericardial effusion, severe myocarditis, etc.; or unstable vital signs requiring vasopressors to maintain blood pressure;
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 UCAR-T cellsA single infusion of CD19 UCAR-T cells will be administered intravenously after lymphodepletion chemotherapy. | Drug | Anti-CD19 UCAR-T cells |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 28 dyas
The incidence of adverse events after CAR-T cell infusion was assessed by CTCAE, version 5.0.
Total improvement score (TIS) after anti-CD19 UCAR-T cells infusion.
Time frame: 6 months
The total improvement score between 0-100 also corresponds to the degree of improvement, with higher improvement scores indicating greater improvement.
Disease Activity Score (DAS) after anti-CD19 UCAR-T cells infusion.
Time frame: 6 months
Including musculoskeletal system score and skin mucosal score, with a total score of 20 points. The higher the score, the higher the disease activity
Changes of myositis specific antibody levels and serum muscle enzyme levels after anti-CD19 UCAR-T cells infusion.
Time frame: 6 months
Improvement in muscle inflammation infiltration by MRI after anti-CD19 UCAR-T cells infusion.
Time frame: 6 months
Childhood Myositis Assessment Scale (CMAS) score after anti-CD19 UCAR-T cells
Time frame: 6 months
The total score is 52 points, the lower the score, the weaker the muscle strength and the more severe the disease.
4 secondary outcomes
Cmax of anti-CD19 UCAR-T cells [Cell dynamics]
Time frame: 1 month
Tmax of anti-CD19 UCAR-T cells [Cell dynamics]
Time frame: 1 month
AUCs of anti-CD19 UCAR-T cells [Cell dynamics]
Time frame: 1 month
Changes of myth enzyme after anti-CD19 UCAR-T cells
Time frame: 1 year
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 13 Nov 2024.
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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 Anti-CD19 UCAR-T cells in people with Dermatomyositis, Juvenile and Dermatomyositis, run by Chongqing Precision Biotech Co., Ltd 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 16 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 13 Nov 2024. 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 Lu Mei ping, M.D (+86 13685773988, meipinglu@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 28 dyas). The study lists 6 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 13 Nov 2024.