BridgeMD
New treatment option for Refractory Immune-mediated Platelet Transfusion Refractoriness
Official title RN1201 Injection in the Treatment of Antibody-Mediated Diseases
ClinicalTrials.gov ID: NCT07072247
What this study is testing
What is CD19 and BCMA-targeted allogeneic CAR-T cells?
CD19 and BCMA-targeted allogeneic CAR-T cells is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for refractory immune-mediated platelet transfusion refractoriness.
Also referred to as RN1201.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 to 65
You may be able to join if
- Inclusion: - specific for Refractory immune-mediated PTR:
- Aged 16-65 years
- Diagnosed with refractory immune-mediated PTR
- Resistant to at least 3 standard therapies
- Able to understand the study and consent
You likely can't join if
- Exclusion: - specific for Refractory immune-mediated PTR:
- 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)
See the full eligibility criteria
Who can join
- Inclusion: - specific for Refractory immune-mediated PTR:
- 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)
- 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...
- 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...
- 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...
- 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...
What rules you out
- Exclusion: - specific for Refractory immune-mediated PTR:
- 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
- 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 \ 1.5× upper limit of normal (ULN); immunoglobulin G (IgG) \<5 g/L.
- people with Class III or IV heart failure according to the NYHA classification (see Appendix I)
- people 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...
- 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...
- 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...
- Any severe and/or unstable pre-existing medical, psychiatric condition, or other disease that the investigator considers may interfere with the patient's how well it works, safety, informed consent, or compliance with...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Suzhou, Jiangsu, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Suzhou, Jiangsu, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.