Treatment for Immune Thrombocytopenia Using CAR T Cells
Treatment of Refractory Immune Thrombocytopenia
This study is testing a new CAR T cell therapy to see if it can help people with hard-to-treat immune thrombocytopenia by targeting the cells causing their condition.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years to 60 Years |
| Sex | All |
| Sponsor | iCell Gene Therapeutics Industry-sponsored |
| Drugs / interventions | rituximab, belimumab |
| Locations | 1 site (Chengdu, Sichuan) |
| Trial ID | NCT06787989 on ClinicalTrials.gov |
What this trial studies
This phase I interventional study evaluates the safety and tolerability of BCMA-CD19 cCAR T cells in patients suffering from refractory immune thrombocytopenia (ITP) associated with autoimmune diseases. The study aims to address the limitations of current treatments by targeting and depleting the abnormal antibody-producing B cells and plasma cells responsible for the condition. Participants will receive the CAR T cell therapy in an open-label format, allowing for direct observation of treatment effects and side effects. The study is designed to provide insights into a potentially more effective treatment option for patients who have not responded to existing therapies.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 to 60 diagnosed with immune thrombocytopenia associated with autoimmune diseases such as systemic lupus erythematosus or primary Sjogren's syndrome.
Not a fit: Patients with serious accompanying diseases or those who do not meet the specific inclusion criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of refractory immune thrombocytopenia, reducing the risk of life-threatening bleeding and improving patients' quality of life.
How similar studies have performed: While CAR T cell therapies have shown promise in other hematological conditions, this specific application for refractory immune thrombocytopenia is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 1\. Age: 18\~60 years old; 2. Diagnosed with immune thrombocytopenia associated with autoimmune diseases including systemic lupus erythematosus (according to the 1997 or 2009 ACR classification criteria), primary Sjogren's syndrome (according to the 2002 ACR/EULAR international classification criteria), undifferentiated connective tissue disease (according to the 1999 international classification criteria). Or patient without clinical manifestations related to connective tissue disease, but with positive anti nuclear antibodies (≥ 1:100) and/or without positive anti SSA/Ro-52 antibodies;Serum creatinine \<221.0μmol/L (2.5mg/dl); 3. platelet count\<30 × 10 ⁹/L or platelet count ≥ 20 × 10 ⁹/L, accompanied by bleeding symptoms (bleeding symptom score ≥ 2 points). No obvious active infection; 4. Voluntary participation and informed consent signed by the patient or his/her legal/authorized representative. Exclusion Criteria: \- 1. Serious accompanying diseases that researchers consider clinically significant due to poor control, such as (but not limited to) neurological, cardiovascular, renal, liver, endocrine, or gastrointestinal diseases CNS disease: Active central nervous system (CNS) lupus (including epilepsy, psychosis, organic encephalopathy syndrome, cerebrovascular accident \[CVA\], encephalitis or CNS vasculitis), visual Disorders, cranial neuropathy requiring intervention 2. Abnormal liver function: aspartate transaminase (AST) or alanine transaminase (ALT) or glutamyl transpeptidase (GGT) detection value is greater than 1.5 times the upper limit of normal (ULN);; urinary protein quantification\>1g/24h. 3\. History of malignant tumors 4. Active hepatitis B or C.，and HIV positive 5. Individuals with a history of drug allergies or allergies. 6. Have any other clinically significant disease history or current disease that, in the judgment of the research physician, may pose a risk to the safety of the subjects, or interfere with the completion of the research procedure and the evaluation of safety and efficacy.
Where this trial is running
Chengdu, Sichuan
- West China Hospital — Chengdu, Sichuan, China (Recruiting)
Study contacts
- Study coordinator: Qibing Xie, MD
- Email: xieqibing1971@163.com
- Phone: 0118618980601299
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.