Using mesenchymal stem cells to treat refractory immune thrombocytopenia

Single-arm, Single-center Clinical Study of Mesenchymal Stem Cells in the Treatment of Refractory Primary Immune Thrombocytopenia

This study is testing whether giving mesenchymal stem cells to people with hard-to-treat immune thrombocytopenia can help raise their platelet counts.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 4 Years to 75 Years
• Sex: All
• Sponsor: Guangzhou Bio-gene Technology Co., Ltd (Industry-sponsored)
• Drugs / interventions: rituximab
• Locations: 1 site (Guangdong, Guangdong)
• Trial ID: NCT06813157 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the safety and efficacy of mesenchymal stem cells (MSCs) in patients with refractory immune thrombocytopenia (ITP). Participants, aged 4 to 75, will receive five infusions of MSCs over a treatment period, with follow-ups to assess outcomes at various intervals. The study involves a single-center approach where subjects are monitored for safety and effectiveness after treatment. The goal is to determine if MSCs can improve platelet counts in patients who have not responded to standard therapies.

Who should consider this trial

Good fit: Ideal candidates are patients aged 4 to 75 with a clinical diagnosis of refractory ITP who have not responded to first-line and second-line treatments.

Not a fit: Patients who have not been diagnosed with ITP or those who have not exhausted other treatment options may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with refractory ITP, potentially improving their platelet counts and overall health.

How similar studies have performed: While the use of MSCs in treating various conditions is being explored, this specific application for refractory ITP is relatively novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

1. The subjects voluntarily signed the informed consent form;
2. The age is from 4 to 75 years old (including the critical value), male or female;
3. Patients clinically diagnosed as ITP (meeting the diagnostic criteria of the Chinese guidelines for the diagnosis and treatment of Primary immune Thrombocytopenia in Adults (2020 Edition)) had persistent thrombocytopenia for more than 3 months, but were ineffective to first-line drugs, second-line drugs for platelet production and rituximab, or ineffective to splenectomy / recurrence after operation.
4. Patients who received other maintenance regimens (including but not limited to corticosteroids, azathioprine, danazol or mycophenolate mofetil), but the stable dose had been maintained for at least 4 weeks, and the dose should remain unchanged during the trial period;
5. During screening, the liver and kidney function of the subjects met the following criteria: alanine aminotransferase (ALT) and / or aspartate aminotransferase (AST) ≤ 3 times of the upper limit of the normal value; total bilirubin ≤ 1.5 times of the upper limit of the normal value; creatinine ≤ 1.5 times of the upper limit of the normal value or creatinine clearance rate \& gt; 75ml / min;
6. Women of childbearing age must have negative blood or urine pregnancy tests and are not breast-feeding, and agree to use medically approved contraceptive measures (such as intrauterine devices, condoms or contraceptive) during the study period.
7. Fertile male patients must agree to use barrier contraception or physical abstinence during the study;
8. The subjects were able to understand the nature and purpose of the trial, including possible risks and side effects, and to understand the researchers' oral and written medical orders and comply with the requirements of the trial.

Exclusion Criteria:

1. Have a history of severe allergic diseases or are allergic to research drugs;
2. There may be a history of angina pectoris, myocardial infarction, heart failure, severe arrhythmia, etc.
3. Combined use of anticoagulants or antiplatelet drugs;
4. In the first study, he was treated with gamma globulin within 2 weeks before medication.
5. The first study received rituximab within 24 weeks before treatment;
6. Other clinical trial drugs were used within 1 month before the use of drugs in the first study.
7. Uncontrolled hypertension (blood pressure is still higher than 160/100mmHg after active treatment);
8. Have a history of malignant tumor;
9. Those with a history of hepatitis B, or hepatitis C, or HIV, or immunodeficiency, or positive results of laboratory tests (hepatitis B surface antigen, or hepatitis C antibody, or HIV antibody) during screening;
10. Active / latent tuberculosis infection or other pathogen infection;
11. Live attenuated vaccine was vaccinated within 1 month before drug use in the first study, or there was a live attenuated vaccine program during the study period;
12. Hypogammaglobulinemia (IgG\<400mg/dl) or IgA deficiency (IgA\<10mg/dl);
13. History of important organ transplantation;
14. Patients with a history of severe mental illness;
15. The researchers believe that it is not appropriate for patients to participate in other conditions of this trial.

Where this trial is running

Guangdong, Guangdong

• Dongguan Taixin Hospital — Guangdong, Guangdong, China (Recruiting)

Study contacts

• Study coordinator: Chaoke Bu, Doctor

Conditions ITP - Immune Thrombocytopenia