Refractory Immune-mediated Platelet Transfusion RefractorinessPhase 1NCT07072247
RN1201 Injection in the Treatment of Antibody-Mediated Diseases
A Phase 1 study testing CD19 and BCMA-targeted allogeneic CAR-T cells in people with Refractory Immune-mediated Platelet Transfusion Refractoriness and Relapsed or Refractory Immune Thrombocytopenia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
This single-arm, open-label exploratory trial aims to evaluate the safety, feasibility, and preliminary efficacy of RN1201 Injection in patients with antibody-mediated diseases, including refractory immune-mediated platelet transfusion refractoriness (PTR) and relapsed or refractory immune thrombocytopenia (ITP). Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Refractory Immune-mediated Platelet Transfusion Refractoriness, Relapsed or Refractory Immune Thrombocytopenia
- What is being tested?
- CD19 and BCMA-targeted allogeneic 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
- 7 Jul 2025to 30 Jul 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged 16-65 years
- Diagnosed with refractory immune-mediated PTR
- Resistant to at least 3 standard therapies
- Able to understand the study and consent
- Projected survival time exceeding three months
- Left Ventricular Ejection Fraction (LVEF) ≥0.5 (as measured by echocardiogram)
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- Creatinine <1.6 mg/dL
- Aspartate Aminotransferase (AST) < three times the upper limit of normal
- Total bilirubin <2.0 mg/dL
- Karnofsky Performance Status (KPS) score ≥60.
- Inclusion: -specific for Relapsed or Refractory Immune Thrombocytopenia
- Written informed consent obtained;
- Male or female patients aged 18 years or older on the day of informed consent signing;
- Left Ventricular Ejection Fraction (LVEF) ≥50% with no pericardial effusion;
- As assessed by the investigator, systemic treatment drugs (excluding supportive and symptomatic treatment, and prednisone at a daily dose of ≤10 mg or equivalent) can be discontinued prior to lymphodepletion preconditioning.
- Historically diagnosed with primary immune thrombocytopenia (ITP) (based on the 2019 International Working Group for ITP and the American Society of Hematology (ASH));
- At least two consecutive blood routine examinations showing reduced platelet count, with no significant abnormalities in blood cell morphology on peripheral blood smear microscopy;
- At the screening visit, the subject has no splenomegaly;
- Bone marrow examination: the bone marrow cytology of ITP patients is characterized by increased or normal megakaryocytes with maturation disorders (investigators may assess whether to accept previous bone marrow examination reports, and if previous reports are used, they must be kept as copies in the study documents);
- The subject is a refractory ITP patient who has not responded to first-line treatment drugs, second-line thrombopoiesis-stimulating agents, and rituximab therapy, or who has undergone ineffective splenectomy or postoperative recurrence, and after re-evaluation of the diagnosis, is still confirmed to have ITP;
- Relapsed ITP is defined as a decrease in platelet count to below 30×10⁹/L after an initial response to treatment, or to less than twice the baseline level, or the reappearance of bleeding symptoms.
- The subject has received at least four weeks of the most recent treatment (non-biological background therapy, antimalarial monotherapy, antimalarial combined with oral glucocorticoids (OCS) and/or immunosuppressants, or combined therapy with OCS and/or immunosuppressants).
Who cannot join
- Uncontrolled active infection
- Active hepatitis B or C infection
- Patient has HIV or syphilis infection
- Patient is pregnant or breastfeeding
- Patient has a history of allogeneic hematopoietic stem cell transplantation (allo-HSCT)
- Conventional treatment for antibody-mediated disease is effective
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- According to the New York Heart Association (NYHA) classification, patients with Class III/IV cardiovascular dysfunction
- Other contraindications that make participation in this study unsuitable.
- Exclusion: - specific for Relapsed or Refractory Immune Thrombocytopenia
- Patients with the following conditions at the screening visit: Neutrophil count <1×10⁹/L; serum creatinine >1.5× upper limit of normal (ULN); immunoglobulin G (IgG) <5 g/L.
- Subjects with Class III or IV heart failure according to the NYHA classification (see Appendix I)
- Subjects with a history of epilepsy or other central nervous system diseases
- Patients with active viral, bacterial or other infections requiring systemic treatment at the screening visit (including active or latent tuberculosis (TB) or SARS-CoV-2), or with a history of clinically significant recurrent infections (e.g., Bacillus infection);
- Herpes or varicella-zoster virus infection within 12 weeks prior to the screening visit (particularly shingles);
- Patients positive for HCV or HBsAg are excluded. HBcAb-positive patients are eligible only if HBsAg (regardless of anti-HBs status) and HBV DNA are negative;
- Known history of primary or secondary immunodeficiency, or positive test results for HIV (ELISA and Western blot) at the screening visit;
- Live vaccine or attenuated live vaccine administration within 4 weeks prior to the baseline visit;
- Breastfeeding or pregnancy at the screening visit or prior to medication administration (positive serum or urine - β-hCG pregnancy test);
- Females of childbearing potential and males whose partners are of childbearing potential must use medically approved contraception or abstain from sexual intercourse during the study treatment period and for at least 6 months after its completion. Females of childbearing potential must have a negative serum HCG test within 7 days before study enrollment and must not be breastfeeding;
- History of malignancy, except for cured non-melanoma skin cancer, carcinoma in situ (e.g., cervical, breast, bladder, or prostate cancer), and tumors in complete remission for at least 3 years without evidence of recurrence;
- Any severe and/or unstable pre-existing medical, psychiatric condition, or other disease that the investigator considers may interfere with the patient's efficacy, safety, informed consent, or compliance with the trial protocol;
- Known hypersensitivity, intolerance, or contraindication to RN1201 or any excipients in the study drugs (including Fludarabine, Cyclophosphamide, and Tocilizumab), or a history of severe allergic reactions;
- Participation in other investigational studies within 30 days prior to enrollment or within five half-lives of the study drug, whichever is longer.
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
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu
Xiaowen Tang, 13913538266, tangxiaowen@suda.edu.cn
About this study
Official title: An Exploratory Clinical Study on the Safety and Efficacy of RN1201 Injection in the Treatment of Antibody-Mediated Diseases
This single-arm, open-label exploratory trial aims to evaluate the safety, feasibility, and preliminary efficacy of RN1201 Injection in patients with antibody-mediated diseases, including refractory immune-mediated platelet transfusion refractoriness (PTR) and relapsed or refractory immune thrombocytopenia (ITP). Patients will receive RN1201 cells infusion following lymphodepletion. The study will assess safety, response rates, B-cell depletion, and immune reconstitution. Exploratory analyses will examine in vivo persistence and activity of RN1201.
- Conditions
- Refractory Immune-mediated Platelet Transfusion Refractoriness, Relapsed or Refractory Immune Thrombocytopenia
- Being tested
- CD19 and BCMA-targeted allogeneic CAR-T cells
- Medical subject headings
- Recurrence, Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD19 and BCMA-targeted allogeneic CAR-T cellsGenetic
Also called RN1201
Lymphodepletion chemotherapy followed by CD19 and BCMA-targeted allogeneic CAR-T cells infusion
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic CAR-T cell therapy | Experimental | Antibody-Mediated Diseases treated with RN1201 Injection Receives: Genetic: CD19 and BCMA-targeted allogeneic CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence and severity of treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs)
To characterize the safety of RN1201 Cells for Antibody-mediated Diseases
Measured: within 4 weeks after infusion
Secondary outcomes (3)
Objective Response Rate (ORR)
The responses will be assessed based on changes of platelet count and incidence of bleeding events, according to predefined response criteria
Measured: From infusion up to 12 months post-treatment.
Pharmacokinetic (PK) of RN1201
Levels of RN1201 CAR-positive T cells in the blood and/or bone marrow
Measured: From infusion up to 12 months post-treatment.
Pharmacodynamic (PD)-B cell depletion
Levels of B cells (direct and indirect measures) in the blood and/or bone marrow
Measured: From infusion up to 12 months post-treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2025
- Study start7 Jul 2025
- Primary completion30 Jul 2026 (estimated)
- Study completion30 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Immune-mediated Platelet Transfusion RefractorinessStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Allorunning Therapeutics
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07072247
- Sponsor's protocol ID
- RN1201-Ab
- Status last verified
- Jul 2025
- First submitted
- 9 Jul 2025
- Last updated
- 12 Jun 2026
- 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 12 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Clinical Study on the Safety and Efficacy of RN1201 Injection in the Treatment of Antibody-Mediated Diseases
- ClinicalTrials.gov ID
- NCT07072247
- Acronym / sponsor ID
- RN1201-Ab
- Conditions
- Refractory Immune-mediated Platelet Transfusion Refractoriness, Relapsed or Refractory Immune Thrombocytopenia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Collaborators
- Allorunning Therapeutics
- Study start
- 7 Jul 2025 (actual)
- Primary completion
- 30 Jul 2026 (estimated)
- Study completion
- 30 Jul 2027 (estimated)
- First posted
- 18 Jul 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 single-arm, open-label exploratory trial aims to evaluate the safety, feasibility, and preliminary efficacy of RN1201 Injection in patients with antibody-mediated diseases, including refractory immune-mediated platelet transfusion refractoriness (PTR) and relapsed or refractory immune thrombocytopenia (ITP). Patients will receive RN1201 cells infusion following lymphodepletion. The study will assess safety, response rates, B-cell depletion, and immune reconstitution. Exploratory analyses will examine in vivo persistence and activity of RN1201.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Allogeneic CAR-T cell therapy | Experimental | Genetic: CD19 and BCMA-targeted allogeneic CAR-T cellsAntibody-Mediated Diseases treated with RN1201 Injection |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (22 listed)
- Aged 16-65 years
- Diagnosed with refractory immune-mediated PTR
- Resistant to at least 3 standard therapies
- Able to understand the study and consent
- Projected survival time exceeding three months
Key exclusion criteria (23 listed)
- Uncontrolled active infection
- Active hepatitis B or C infection
- Patient has HIV or syphilis infection
- Patient is pregnant or breastfeeding
- Patient has a history of allogeneic hematopoietic stem cell transplantation (allo-HSCT)
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 and BCMA-targeted allogeneic CAR-T cellsAlso: RN1201Lymphodepletion chemotherapy followed by CD19 and BCMA-targeted allogeneic CAR-T cells infusion | Genetic | Allogeneic CAR-T cell therapy |
Primary outcome
The incidence and severity of treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs)
Time frame: within 4 weeks after infusion
To characterize the safety of RN1201 Cells for Antibody-mediated Diseases
3 secondary outcomes
Objective Response Rate (ORR)
Time frame: From infusion up to 12 months post-treatment.
Pharmacokinetic (PK) of RN1201
Time frame: From infusion up to 12 months post-treatment.
Pharmacodynamic (PD)-B cell depletion
Time frame: From infusion up to 12 months post-treatment.
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 12 Jun 2026.
- Individual participant dataSharing planned: No
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 CD19 and BCMA-targeted allogeneic CAR-T cells in people with Refractory Immune-mediated Platelet Transfusion Refractoriness and Relapsed or Refractory Immune Thrombocytopenia, run by The First Affiliated Hospital of Soochow 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 22 inclusion and 23 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 12 Jun 2026. 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 Xiaowen Tang, PhD (+86-13913538266, tangxiaowen@suda.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence and severity of treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs) (time frame: within 4 weeks after infusion). 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 12 Jun 2026.