Gene therapy for patients with relapsed immune thrombocytopenia

An Exploratory Clinical Study of Anti-CD19/BCMA CAR Gene Vector Injection (LCAR1901) for the Treatment of Relapsed and Refractory Immune Thrombocytopenia

This study is testing a one-time gene therapy injection to see if it can help people with relapsed immune thrombocytopenia who haven't had success with other treatments.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Anhui Provincial Hospital (Government)
• Drugs / interventions: rituximab
• Locations: 1 site (Changsha, Hunan)
• Trial ID: NCT06973356 on ClinicalTrials.gov

What this trial studies

This open-label, single-site study involves administering an Anti-CD19/BCMA CAR gene vector injection to up to 18 participants suffering from relapsed and refractory immune thrombocytopenia. The study aims to evaluate the safety and efficacy of this one-time intravenous treatment. Participants must have a clinical diagnosis of primary immune thrombocytopenia and have previously undergone second-line therapies without success. The study will monitor the participants' platelet counts and overall health following the intervention.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a clinical diagnosis of primary immune thrombocytopenia and a platelet count below 30×10^9/L.

Not a fit: Patients who have not been diagnosed with immune thrombocytopenia or those who have not responded to prior second-line therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve platelet counts and reduce bleeding risks for patients with immune thrombocytopenia.

How similar studies have performed: While gene therapy approaches are emerging in the treatment of autoimmune diseases, this specific application for immune thrombocytopenia is relatively novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* 1\. Age≥ 18 years old, regardless of gender. 2. Clinical diagnosis of primary immune thrombocytopenia for at least 6 months, platelet count \< 30×10\^9/L within 48 hours before participating in the study.

  3\. Positive anti-platelet glycoprotein autoantibodies (such as GPIIb/IIIa). 4. Prior second-line ITP therapy (first-line treatment includes: corticosteroids or immunoglobulins; Second-line therapies include thrombopoietin receptor agonists (eg, eltrombopag, romiplostim) and/or rituximab, but are ineffective (platelet count \< 30×10\^9/L after treatment, or platelet count does not increase twice as much as baseline, or there is bleeding), or relapse after effective treatment (platelet count falls below 30×109/L after effective treatment, or falls below baseline, or bleeding symptoms) or is difficult to maintain after discontinuation of TPO agonists.

  5\. Bone marrow examination shows megakaryocytosis or normal. 6. Basic normal functions of important organs:Echocardiography shows an ejection fraction of ≥50% and no significant abnormalities on ECG.Creatinine clearance (CrCl) (Cockcroft-Gault formula) ≥ 30 mL/min.Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3.0 × upper limit of normal (ULN).Total bilirubin (TBIL) and alkaline phosphatase (AKP or ALP) ≤ 2.0× ULN (Gilbert's syndrome ≤3.0×ULN).Absolute lymphocyte count (ALC) ≥ 0.5×10\^9/L; Absolute neutrophil count (ANC) ≥1×10\^9/L; Hemoglobin (Hb) ≥ 60 g/L; Platelet count ≥ 10×10\^9/L.Oxygen saturation \> 92%.ECOG performance status ≤2 7. Males and women of childbearing potential must agree to use effective contraception from the time of signing the informed consent form until 1 year after the use of the study drug. Women of childbearing potential must have a negative blood pregnancy test at screening and prior to drug infusion and must not be breastfeeding.

Exclusion Criteria:

* 1\. Thrombocytopenia caused by myelodysplastic syndrome, early aplastic anemia, atypical aplastic anemia, thrombotic thrombocytopenic purpura, etc.

  2\. During the screening period, bone marrow examination showed myelofibrosis MF≥2 (European consensus scoring standard Thieleja 2005) or bone marrow examination showed the presence of a primary disease other than ITP that can lead to thrombocytopenia.

  3\. History of hypersensitivity to any component of the therapeutic medication. 4. Major organs: NYHA class III to IV congestive heart failure. Myocardial infarction or coronary artery bypass grafting (CABG) or coronary artery stent implantation within 6 months. Ventricular arrhythmias, or history of unexplained syncope (excluding vasovagal syncope or dehydration). History of severe non-ischemic cardiomyopathy.

  5\. Malignant disease within 3 years prior to screening, except for the following: malignant disease that has been curatively treated before enrollment and has no known active disease for 3 years ≥; or well-treated non-melanoma skin cancer with no evidence of disease.

  6\. Symptomatic deep vein thrombosis or pulmonary embolism within the past 6 months or currently requiring anticoagulation.

  7\. Participated in other interventional clinical studies within 1 month prior to screening.

  8\. Vaccination of live attenuated vaccine within 4 weeks prior to screening. 9. Stroke or seizure within 6 months prior to signing the ICF (excluding old lacunar cerebral infarction).

  10\. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and peripheral blood hepatitis B virus (HBV) DNA titer test exceeds the normal range; Hepatitis C virus (HCV) antibody is positive and the hepatitis C virus (HCV) RNA titer in peripheral blood exceeds the normal range; positive for human immunodeficiency virus (HIV) antibodies; Positive syphilis test.

  11\. Known history of bone marrow stem cell disease 12. Other conditions that the investigators consider unsuitable to participate in the study.

Where this trial is running

Changsha, Hunan

• Hunan Siweikang Therapeutic Co.Ltd — Changsha, Hunan, China (Recruiting)

Study contacts

• Principal investigator: bing xing wang, M.D — The First Affiliated Hospital of University of Science and Technology of China
• Study coordinator: bing xing wang, M.D

Conditions Immune Thrombocytopenia; immune thrombocytopenia; Autoimmune diseases