Anti-BCMA CAR T-Cell Therapy for R/R ITP

NCT05315778 | Unknown Phase 2

• 1 other identifier: BCRITP
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, single-center, open-label, single-arm study, to evaluate the efficacy and safety of Anti-BCMA chimeric antigen receptor T cell therapy（BCMA CAR-T）for patients with relapse/refractory Immune thrombocytopenia(R/R ITP).

Conditions

ITP

Keywords

CAR-T; BCMA

Outcome Measures

Primary Outcomes (1)

• overall response

  The number of participants who achieved CR (defined as platelet count≥100x10e9/L) and PR (defined as platelet count ≥30x10e9/L and at least a 2-fold increase the baseline count and absence of bleeding) at follow-up.

  6-months

Secondary Outcomes (6)

• Time to response

  6-months

• Duration of response

  6-months

• Incidence of adverse events

  6-months

• Evaluation of bleeding events

  6-months

• Evaluation of concomitant therapy

  6-months

Study Arms (1)

Anti-BCMA CAR T-cells infusion

EXPERIMENTAL

R/R ITP patients will accept infusion of autologous anti-BCMA CAR T-cells with a total of 1.0-2.0×10e7/Kg. The patients will be follow-up for 6 months post CAR T-cell therapy.

Biological: autologous anti-BCMA chimeric antigen receptor T cells

Interventions

autologous anti-BCMA chimeric antigen receptor T cells BIOLOGICAL

Lymphoadenodepletion chemotherapy with FC (fludarabine 30mg/ m2 for 3 consecutive days and cyclophosphamide 300mg/m2 for 3 consecutive days) will be given at day -5, -4 and -3 before CAR T-cells infusion. A total of 1.0-2.0×10e7/Kg autologous anti-BCMA CAR T-cells will be infused by dose-escalation after the lymphoadenodepletion chemotherapy. Dose of CAR T-cells are allowed to be adjusted according to the severity of cytokine release syndrome.

Anti-BCMA CAR T-cells infusion

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Refractory ITP defined according to the recent consensual criteria ( 'Chinese guideline on the diagnosis and management of adult primary immune thrombocytopenia (version 2020)'), or relapse ITP defined as ITP patients who have responded to first-line therapy (glucocorticoids or immunoglobulins) and anti-CD20 monoclonal antibody, but cannot maintain the response.
• Ages 18-65 years inclusive.
• Adequate venous access for apheresis or venous blood and no other contraindications for leukocytosis.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
• Subjects should have full capacity for civil conduct, understand necessary information，sign the informed consent form voluntarily，and have good corporation with the content of this research protocol.

You may not qualify if:

• Secondary ITP.
• Patients with a known history or prior diagnosis of arterial thrombosis (such as cerebral thrombosis, myocardial infarction, etc.), or comorbidity of venous thrombosis (such as deep vein thrombosis, pulmonary embolism), or are using anticoagulant/antiplatelet drug at the beginning of trial.
• Patients with a known history or prior diagnosis of serious cardiovascular disease.
• Patients with uncontrolled infection, organ dysfunction or any uncontrolled active medical disorder that would preclude participation as outlined.
• Patients with malignancy or history of malignancy.
• Failed T cell expansion test.
• During screening, hemoglobin \<100g/L; absolute value of neutrophil count \<1.5×10\^9/L.
• During screening, serum creatinine concentration \> 1.5x the upper limit of the normal range, total bilirubin \> 1.5x the upper limit of the normal range, alanine aminotransferase and aspartate aminotransferase \> 3x the upper limit of the normal range, Left ventricular ejection fraction ≤ 50% by echocardiography, Pulmonary function ≥ grade 1 dyspnea (CTCAE v5.0), blood oxygen saturation\<91% without oxygen inhalation.
• Prothrombin time (PT) or prothrombin time-international normalized ratio (PT-INR) or activated partial thromboplastin time (APTT) exceeding 20% of the normal reference range; or a history of coagulation abnormalities other than ITP.
• Either HIV antibody or syphilis antibody is positive; hepatitis C antibody is positive and the detection of HCV-RNA exceeds the laboratory test upper reference limit; hepatitis B surface antigen is positive and the detection of HBV-DNA exceeds the laboratory test upper reference limit.
• Participated in other clinical studies within 3 months before this CAR-T cell infusion.
• Patients is pregnant or breastfeeding, or planning pregnancy.
• Patients is fertile and the investigator determines the case is inappropriate to participate.
• History of severe drug allergy or known allergy to CAR-T treatment related drugs.
• Suspected or established alcohol, drug or drug abuse.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead
• Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd: collaborator

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

RECRUITING

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2022
• First Posted: April 7, 2022
• Study Start: January 1, 2022
• Primary Completion: December 31, 2022
• Study Completion: June 30, 2023
• Last Updated: April 7, 2022

Record last verified: 2022-03

Locations

CN (1)