Juvenile DermatomyositisPhase 1NCT06569472
Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis
A Phase 1 study testing CD19-targetd CAR-T in people with Juvenile Dermatomyositis, run by Chongqing Precision Biotech Co., Ltd at 1 site in China.
Trial summary
This is a Phase I clinical trial to evaluate the efficacy and safety of CD19-targeted CAR-T in the treatment of refractory juvenile dermatomyositis (RJDM).The experiment was divided into two phases: dose exploration (Part A) and dose extension (Part B). Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Juvenile Dermatomyositis
- What is being tested?
- CD19-targetd CAR-T
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 2 Jul 2024to 31 Jul 2028
- 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 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age: ≥5 years and <17 years old
- To meet the diagnostic criteria of JDM, four or five of the following criteria must be met:① symmetrical proximal muscle weakness; ②Characteristic skin changes, including positive dermatitis (purplish red rash on upper eyelid with periorbital edema) and Gottron papules (red patchy squamous papules on the back of knuckles); ③ The level of one muscle enzyme in serum was increased; ④ Positive myositis antibody; ⑤Electromyography shows denervation and myopathy; ⑥ Muscle biopsies showed necrosis and inflammation.
- The classification criteria of RJDM must meet ① and any of the criteria②-④: ① Patients who are intolerant or unresponsive to glucocorticoids and at least 2 immunosuppressants, adequate hormone therapy and duration of 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.
- myositis specific antibody positive, defined as MDA-5, NXP2, TIF-1γ, Ro-52 and any other positive;
- If the patient has SRP or HMGCR antibody positive immune-mediated necrotizing myopathy equivalent to RJDM, the inclusion criteria of (2) - (4) can be met.
- The functions of important organs are basically normal:
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- ① Cardiac function: left ventricular ejection fraction (LVEF) ≥55%, no obvious abnormality in electrocardiogram;
- ② Renal function: eGFR≥30ML/min/1.73m2;
- ③ Liver function: AST and ALT≤3.0 ULN, total bilirubin ≤2.0×ULN;
- ④ Lung function: Lung function is basically normal, SpO2≥92%;
- Have the criteria for simple or intravenous blood collection, and no other contraindications for cell collection;
- 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 this clinical trial and signs an informed consent indicating that he/she understands the purpose and procedure of this clinical trial and is willing to participate in the study.
Who cannot join
- Had previously received CAR T cell therapy;
- Have other autoimmune or rheumatic diseases other than JDM;
- primary immunodeficiency or severe secondary immunodeficiency that has not been corrected;
- accompanied by serious infectious diseases, including but not limited to active tuberculosis, latent tuberculosis infection, active viral hepatitis, etc.;
- Evidence of active malignant disease or diagnosis of malignant tumor (including hematological malignancies and solid tumors, except resected and cured skin basal cell carcinoma)
- 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 need hypertensive drugs to maintain their blood pressure;
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- suffering from other diseases that require long-term use of glucocorticoids or immunosuppressants;
- There is an active or uncontrollable infection that requires systemic treatment within 1 week prior to screening;
- 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;
- Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer greater than the normal reference value range; Or hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Or positive for human immunodeficiency virus (HIV) antibodies; Or syphilis test positive; Or cytomegalovirus (CMV) DNA test positive;
- Had received live vaccine within 4 weeks prior to screening;
- Positive blood pregnancy test;
- Patients with known malignant diseases such as tumors before screening;
- Patients who had participated in other clinical trials within 3 months prior to enrollment;
- Situations in which other investigators consider it inappropriate to participate 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
Meiping Lu, M.D, 13685773988, meipinglu@zju.edu.cn
About this study
Official title: Clinical Study of CD19 Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
This is a Phase I clinical trial to evaluate the efficacy and safety of CD19-targeted CAR-T in the treatment of refractory juvenile dermatomyositis (RJDM).The experiment was divided into two phases: dose exploration (Part A) and dose extension (Part B).
Read the detailed description
juvenile dermatomyositis (JDM) is a non-suppurative chronic autoimmune disease and the most common type of juvenile idiopathic inflammatory myopathy.Despite the active treatment measures, some JDM patients are still intolerant or unresponsive to the treatment, resulting in a high disability and mortality rate.Because CD19 is widely expressed on the surface of B lymphocytes, CD19 CAR-T can also cause deep depletion of other CD19+ B cells while killing cancer cells, which is expected to achieve immune reconstruction in patients with autoimmune diseases, and completely change the status quo that such patients need to take hormones and immunosuppressants for a long time.
- Conditions
- Juvenile Dermatomyositis
- Being tested
- CD19-targetd CAR-T
- Keywords
- CAR-T, CD19
- Medical subject headings
- Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD19-targetd CAR-TBiological
Subjects underwent lymphocyte clearance chemotherapy and then received a single intravenous cell infusion
| Study group | Role | What they receive |
|---|---|---|
| CD19-targeted CAR-T | Experimental | The experiment was divided into two phases: dose exploration (Part A) and dose extension (Part B). Receives: Biological: CD19-targetd CAR-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety of CAR-T in the treatment of refractory juvenile dermatomyositis [Safety and Tolerability]
The incidence of adverse events after CAR-T cell infusion was assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, version 5.0).including the type, frequency and severity of adverse events
Measured: 28 days
To evaluate the efficacy of CAR-T in the treatment of refractory juvenile dermatomyositis [Effectiveness]
Disease improvement rate: The number of subjects who achieved disease improvement as a percentage of all subjects who received transfusions.
Measured: 2month,3months
Duration of Response (DOR) of CAR-T treatment in the treatment of refractory juvenile dermatomyositis [Effectiveness]
DOR will be assessed from the first assessment of remission to the first assessment of recurrence or progression of the disease or death from any cause
Measured: 2 years
Secondary outcomes (3)
AUCS of CD19 CAR-T cells [Cell dynamics]
AUCS is defined as the area under the curve in 90 days
Measured: 3 months
CMAX of CD19 CAR-T cells [Cell dynamics]
CMAX is defined as the highest concentration of CEA CAR-T cells expanded in peripheral blood
Measured: 3 months
TMAX of CD19 CAR-T cells[Cell dynamics]
TMAX is defined as the time to reach the highest concentration
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2024
- Study start2 Jul 2024
- Primary completion31 Jul 2027 (estimated)
- Study completion31 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Juvenile DermatomyositisStudies 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
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chongqing Precision Biotech Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Meiping Lu, M.D, Principal investigator, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06569472
- Sponsor's protocol ID
- PBC068
- Status last verified
- Jul 2024
- First submitted
- 21 Aug 2024
- Last updated
- 26 Aug 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2024. 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 Juvenile Dermatomyositis (RJDM)
- ClinicalTrials.gov ID
- NCT06569472
- Acronym / sponsor ID
- PBC068
- Conditions
- Juvenile Dermatomyositis
- Keywords
- CAR-T, CD19
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Chongqing Precision Biotech Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 2 Jul 2024 (actual)
- Primary completion
- 31 Jul 2027 (estimated)
- Study completion
- 31 Jul 2028 (estimated)
- First posted
- 26 Aug 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 is a Phase I clinical trial to evaluate the efficacy and safety of CD19-targeted CAR-T in the treatment of refractory juvenile dermatomyositis (RJDM).The experiment was divided into two phases: dose exploration (Part A) and dose extension (Part B).
Detailed description
juvenile dermatomyositis (JDM) is a non-suppurative chronic autoimmune disease and the most common type of juvenile idiopathic inflammatory myopathy.Despite the active treatment measures, some JDM patients are still intolerant or unresponsive to the treatment, resulting in a high disability and mortality rate.Because CD19 is widely expressed on the surface of B lymphocytes, CD19 CAR-T can also cause deep depletion of other CD19+ B cells while killing cancer cells, which is expected to achieve immune reconstruction in patients with autoimmune diseases, and completely change the status quo that such patients need to take hormones and immunosuppressants for a long time.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD19-targeted CAR-T | Experimental | Biological: CD19-targetd CAR-TThe experiment was divided into two phases: dose exploration (Part A) and dose extension (Part B). |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (13 listed)
- Age: ≥5 years and <17 years old
- To meet the diagnostic criteria of JDM, four or five of the following criteria must be met:① symmetrical proximal muscle weakness; ②Characteristic skin changes, including positive dermatitis (purplish red rash on upper eyelid with periorbital edema) and Gottron papules (red patchy squamous papules on the back of knuckles); ③ The level of one muscle enzyme in serum was increased; ④ Positive myositis antibody; ⑤Electromyography shows denervation and myopathy; ⑥ Muscle biopsies showed necrosis and inflammation.
- The classification criteria of RJDM must meet ① and any of the criteria②-④: ① Patients who are intolerant or unresponsive to glucocorticoids and at least 2 immunosuppressants, adequate hormone therapy and duration of 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.
- myositis specific antibody positive, defined as MDA-5, NXP2, TIF-1γ, Ro-52 and any other positive;
- If the patient has SRP or HMGCR antibody positive immune-mediated necrotizing myopathy equivalent to RJDM, the inclusion criteria of (2) - (4) can be met.
Key exclusion criteria (15 listed)
- Had previously received CAR T cell therapy;
- Have other autoimmune or rheumatic diseases other than JDM;
- primary immunodeficiency or severe secondary immunodeficiency that has not been corrected;
- accompanied by serious infectious diseases, including but not limited to active tuberculosis, latent tuberculosis infection, active viral hepatitis, etc.;
- Evidence of active malignant disease or diagnosis of malignant tumor (including hematological malignancies and solid tumors, except resected and cured skin basal cell carcinoma)
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 |
|---|---|---|
| CD19-targetd CAR-TSubjects underwent lymphocyte clearance chemotherapy and then received a single intravenous cell infusion | Biological | CD19-targeted CAR-T |
Primary outcome
To evaluate the safety of CAR-T in the treatment of refractory juvenile dermatomyositis [Safety and Tolerability]
Time frame: 28 days
The incidence of adverse events after CAR-T cell infusion was assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, version 5.0).including the type, frequency and severity of adverse events
To evaluate the efficacy of CAR-T in the treatment of refractory juvenile dermatomyositis [Effectiveness]
Time frame: 2month,3months
Disease improvement rate: The number of subjects who achieved disease improvement as a percentage of all subjects who received transfusions.
Duration of Response (DOR) of CAR-T treatment in the treatment of refractory juvenile dermatomyositis [Effectiveness]
Time frame: 2 years
DOR will be assessed from the first assessment of remission to the first assessment of recurrence or progression of the disease or death from any cause
3 secondary outcomes
AUCS of CD19 CAR-T cells [Cell dynamics]
Time frame: 3 months
CMAX of CD19 CAR-T cells [Cell dynamics]
Time frame: 3 months
TMAX of CD19 CAR-T cells[Cell dynamics]
Time frame: 3 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 26 Aug 2024.
Related trials for Juvenile Dermatomyositis
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Natural History of Inflammatory Muscle Diseases
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Environmental Risk Factors for the Anti-synthetase Syndrome
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RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
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Adult and Juvenile Myositis
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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.
Meiping Lu, M.D principal investigator
The Children's Hospital of Zhejiang University School of Medicine
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 CD19-targetd CAR-T in people with Juvenile 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 13 inclusion and 15 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 26 Aug 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 Meiping Lu, M.D (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?
To evaluate the safety of CAR-T in the treatment of refractory juvenile dermatomyositis [Safety and Tolerability] (time frame: 28 days). The study lists 3 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 26 Aug 2024.