Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic Diseases (NCT07184450) | Clinical Trial Compass
RecruitingPhase 1
Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic Diseases
China11 participantsStarted 2025-09-01
Plain-language summary
This is an investigator-initiated trial to evaluate the efficacy and safety of BCMA/CD70-targeted CAR-T in the treatment of refractory pediatric rheumatic diseases.
Who can participate
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Diagnosed as juvenile dermatomyositis(JDM) according to the criteria of Bohan and Peter, and meeting the following conditions:
. The classification criteria of RJDM must meet (1) and any one of (2)-(5): (1) Patients who are intolerant or unresponsive to glucocorticoids and at least 2 immunosuppressants, and the duration of adequate hormone therapy should be at least 6 months; (2) The disease progresses rapidly and/or involves organs such as lungs, heart and gastrointestinal tract; (3) Calcification of subcutaneous or muscle and joint tissues; (4) Repeated rashes or skin ulcers; (5) Repeated or persistent myasthenia(muscle MRI indicates extensive, diffuse edema or the Childhood Myositis Assessment Scale(CMAS) should be less than 48 points, and at least two of the following five core measurement indicators should have abnormal results: Physician Global Assessment(PhGA) ≥2cm, Patient Global Assessment(PtGA) ≥2cm, Disease Activity Score(DAS) ≥2 points, Childhood Health Assessment Questionnaire(C-HAQ) ≥0.25 points, muscle enzyme level \> 1.5×upper limit of normal);
. RJDM with anti-synthetase syndrome who are positive for anti-synthetase antibody and those with immune-mediated necrotizing myopathy who are positive for SRP or HMGCR antibody can be included.
. Meet the classification criteria for polyarticular juvenile idiopathic arthritis as defined by the International League of Associations for Rheumatology(ILAR) classification in 2001, and meeting the following conditions: After at least 6 months of traditional DMARDS treatment and at least one stable dose of DMARDS or biologic agent for ≥12 weeks, the disease is still active, that is, there are at least 2 active joints (defined as swollen joints; if there is no swelling, there must be limited passive range of motion, accompanied by pain during movement or joint tenderness).
. Meet the classification criteria for Systemic sclerosis (SSc) as defined by the 2013ACR/EULAR standards, and meeting the following conditions:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To evaluate the safety of CAR-T in the treatment of refractory pediatric rheumatic diseases [Safety and Tolerability]
Timeframe: 28 days
2
The Total Improvement Score (TIS) of CAR-T in the treatment of refractory juvenile dermatomyositis [Effectiveness]
Timeframe: 6 months
3
Minimal disease activity, inactive disease and remission of CAR-T in the treatment of refractory polyarticular juvenile idiopathic arthritis [Effectiveness]
Timeframe: 6 months
4
Ped ACR 30/50/70/90/100 of CAR-T in the treatment of refractory polyarticular juvenile idiopathic arthritis [Effectiveness]
Timeframe: 6 months
5
mRSS of CAR-T in the treatment of refractory systemic sclerosis [Effectiveness]
Timeframe: 6 months
6
MDAI of CAR-T in the treatment of refractory systemic sclerosis [Effectiveness]
Timeframe: 6 months
7
EUSTAR activity index of CAR-T in the treatment of refractory systemic sclerosis [Effectiveness]
. 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,mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, and biologics including rituximab, beliumab, and telitacicept, 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;
. Meet the classification criteria for primary Sjogren's syndrome as defined by the 2002 ACEG classification criteria /2016 EULAR/ACR classification criteria, and meeting the following conditions:
Timeframe: 6 months
8
CRISS of CAR-T in the treatment of refractory systemic sclerosis [Effectiveness]
Timeframe: 6 months
9
Minimal disease activity, inactive disease and remission of CAR-T in the treatment of refractory primary Sjogren's syndrome [Effectiveness]
Timeframe: 6 months
10
The STAR response rate of CAR-T in the treatment of refractory primary Sjogren's syndrome [Effectiveness]
Timeframe: 6 months
11
ESSDAI of CAR-T in the treatment of refractory primary Sjogren's syndrome [Effectiveness]
Timeframe: 6 months
12
clinESSDAI of CAR-T in the treatment of refractory primary Sjogren's syndrome [Effectiveness]
Timeframe: 6 months
13
ESSPRI of CAR-T in the treatment of refractory primary Sjogren's syndrome [Effectiveness]