Idiopathic Inflammatory MyopathiesEarly Phase 1NCT06986018
Clinical Study on the Targeted CD19 Universal CAR-T Cell Injection (RD06-04) for the Treatment of IIM and AAV
A Early Phase 1 study testing RD06-04 Cell Injection Infusion in people with Idiopathic Inflammatory Myopathies and ANCA-Associated Vasculitis, run by Peking University People's Hospital at 1 site in China.
Trial summary
This is an open-label, investigator-initiated clinical trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-04 in patients with refractory IIM and AAV. The study plans to enroll a total of 12 participants, with 6 cases each for IIM and AAV. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Idiopathic Inflammatory Myopathies, ANCA-Associated Vasculitis
- What is being tested?
- RD06-04 Cell Injection Infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 14 Jun 2025to 31 Dec 2026
- Phase
- Early 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- As determined by the investigator, the primary diagnosis is a rheumatic autoimmune disease other than the disease under study, which the investigator believes may confound the efficacy evaluation of the study disease.
- Clinically significant central nervous system disease or pathological changes not caused by the non-study disease within 12 months prior to screening.
- History of allogeneic bone marrow or stem cell transplantation or solid organ transplantation (such as kidney, lung, heart, liver) or plans for such transplantation in the future.
- For IIM patients: Presence of severe rhabdomyolysis or CK levels ≥120×ULN at screening.
- History of, or current significant cardiovascular dysfunction.
- History of malignancy within 5 years prior to signing the ICF.
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- Pregnant or breastfeeding women.
- History of recurrent infections requiring hospitalization and intravenous antibiotics (e.g., three or more episodes of the same type of infection within the past year).
- Positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis B core antibody (HBcAb) with detectable hepatitis B virus (HBV) DNA in peripheral blood; positive for hepatitis C virus (HCV) antibody with detectable HCV RNA in peripheral blood; positive for human immunodeficiency virus (HIV) antibody; positive for syphilis antibody.
- History of drug or alcohol abuse within 1 year prior to screening.
- Any condition that, in the investigator's opinion, may affect study participation, pose a safety risk to the patient, or potentially confound the interpretation of study results.
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
Peking University People's Hospital
Beijing, 100032
Zhanguo Li, Doctor, 010-88324073, li99@bjmu.edu.cn
About this study
Official title: Clinical Study on the Safety, Efficacy, and Pharmacokinetics of a Universal CD19-Targeted CAR-T Cell Injection (RD06-04) in the Treatment of Patients With Refractory Inflammatory Myopathy and ANCA-Associated Vasculitis
This is an open-label, investigator-initiated clinical trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-04 in patients with refractory IIM and AAV. The study plans to enroll a total of 12 participants, with 6 cases each for IIM and AAV. Enrollment for both diseases will proceed in parallel. The dose will be 6×10^6 CAR+T cells/kg (±30%), and patients will receive a single infusion of RD06-04.
- Being tested
- RD06-04 Cell Injection Infusion
- Keywords
- CD19-Targeted Universal CAR-T
- Medical subject headings
- Myositis, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RD06-04 Cell Injection InfusionDrug
CAR T-cell therapy administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.
| Study group | Role | What they receive |
|---|---|---|
| RD06-04 Cell Injection | Experimental | Not described Receives: Drug: RD06-04 Cell Injection Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (TEAE), Serious Adverse Events (SAE), Adverse Events of Special Interest (AESI)
Measured: Up to 2 years
Secondary outcomes (9)
The proportion of patients with kidney involvement achieving complete kidney remission (CRR)
Measured: At weeks 12, and months 6, 12, 18, and 24 following CAR-T infusion.
The change in UPCR from baseline in patients with kidney involvement.
Measured: At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.
The change in eGFR (estimated glomerular filtration rate) from baseline in patients with kidney involvement.
Measured: At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.
The change in BVAS score from baseline in AAV patients
Birmingham Vasculitis Activity Score (BVAS) has a minimum score of 0 and a theoretical maximum score exceeding 100 (depending on the number and severity of affected organ systems).
A higher score indicates stronger disease activity and a worse prognosis, while a decrease in score (especially to <5) typically suggests effective treatment.
Measured: At 6, 12, 18, and 24 months following CAR-T infusion.
IIM patients were assessed for major clinical remission (Total Improvement Score, TIS) according to the 2016 ACR /EULAR criteria for myocarditis remission.
Total Improvement Score (TIS) has a minimum score of 0 (indicating no improvement) and a maximum score of 100 (indicating maximal improvement).
A higher score represents more significant disease improvement and better treatment efficacy.
Measured: At 6, 12, 18, and 24 months following CAR-T infusion.
The change in FACIT-Fatigue from baseline.
Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) has a minimum score of 0 and a maximum score of 52.
A higher score indicates less severe fatigue and better quality of life.
Measured: At 6, 12, 18, and 24 months following CAR-T infusion.
Assessment of the peak amplification level (Cmax), area under the concentration-time curve (AUC0-28), and persistence profile of RD06-04.
Measured: Up to 2 years
The change in HAQ-DI from baseline.
Health Assessment Questionnaire-Disability Index (HAQ-DI) has a minimum score of 0 and a maximum score of 3.
A higher score indicates more severe physical dysfunction and worse quality of life.
Measured: At 6, 12, 18, and 24 months following CAR-T infusion.
Assessment of the incidence and titer levels of anti-RD06-04 specific anti-drug antibodies (ADA).
Measured: 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 posted22 May 2025
- Study start14 Jun 2025 (estimated)
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Inflammatory MyopathiesStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe 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
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhanguo Li, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06986018
- Sponsor's protocol ID
- BHCT-RD06-04-08
- Status last verified
- Apr 2025
- First submitted
- 13 Apr 2025
- Last updated
- 22 May 2025
- Results posted
- No
- Data monitoring committee
- No
- 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 22 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on the Safety, Efficacy, and Pharmacokinetics of a Universal CD19-Targeted CAR-T Cell Injection (RD06-04) in the Treatment of Patients With Refractory Inflammatory Myopathy and ANCA-Associated Vasculitis
- ClinicalTrials.gov ID
- NCT06986018
- Acronym / sponsor ID
- BHCT-RD06-04-08
- Keywords
- CD19-Targeted Universal CAR-T
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 14 Jun 2025 (estimated)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 22 May 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 open-label, investigator-initiated clinical trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-04 in patients with refractory IIM and AAV. The study plans to enroll a total of 12 participants, with 6 cases each for IIM and AAV. Enrollment for both diseases will proceed in parallel. The dose will be 6×10^6 CAR+T cells/kg (±30%), and patients will receive a single infusion of RD06-04.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RD06-04 Cell Injection | Experimental | Drug: RD06-04 Cell Injection Infusion |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (11 listed)
- As determined by the investigator, the primary diagnosis is a rheumatic autoimmune disease other than the disease under study, which the investigator believes may confound the efficacy evaluation of the study disease.
- Clinically significant central nervous system disease or pathological changes not caused by the non-study disease within 12 months prior to screening.
- History of allogeneic bone marrow or stem cell transplantation or solid organ transplantation (such as kidney, lung, heart, liver) or plans for such transplantation in the future.
- For IIM patients: Presence of severe rhabdomyolysis or CK levels ≥120×ULN at screening.
- History of, or current significant cardiovascular dysfunction.
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 |
|---|---|---|
| RD06-04 Cell Injection InfusionCAR T-cell therapy administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide. | Drug | RD06-04 Cell Injection |
Primary outcome
Adverse Events (TEAE), Serious Adverse Events (SAE), Adverse Events of Special Interest (AESI)
Time frame: Up to 2 years
9 secondary outcomes
The proportion of patients with kidney involvement achieving complete kidney remission (CRR)
Time frame: At weeks 12, and months 6, 12, 18, and 24 following CAR-T infusion.
The change in UPCR from baseline in patients with kidney involvement.
Time frame: At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.
The change in eGFR (estimated glomerular filtration rate) from baseline in patients with kidney involvement.
Time frame: At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.
The change in BVAS score from baseline in AAV patients
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
IIM patients were assessed for major clinical remission (Total Improvement Score, TIS) according to the 2016 ACR /EULAR criteria for myocarditis remission.
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
The change in FACIT-Fatigue from baseline.
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
Assessment of the peak amplification level (Cmax), area under the concentration-time curve (AUC0-28), and persistence profile of RD06-04.
Time frame: Up to 2 years
The change in HAQ-DI from baseline.
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
Assessment of the incidence and titer levels of anti-RD06-04 specific anti-drug antibodies (ADA).
Time frame: Up to 2 years
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 22 May 2025.
- Individual participant dataSharing planned: No
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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 Early Phase 1 study testing RD06-04 Cell Injection Infusion in people with Idiopathic Inflammatory Myopathies and ANCA-Associated Vasculitis, run by Peking University People's Hospital 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 0 inclusion and 11 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: opening soon, as of the sponsor's last update on 22 May 2025. 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?
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 ZhanGuo Li, Doctor (010-88324073, 1i99@bjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (TEAE), Serious Adverse Events (SAE), Adverse Events of Special Interest (AESI) (time frame: Up to 2 years). 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 22 May 2025.