A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis

Early Phase 1

Recruiting

• Conditions: POEMS Syndrome; Autoimmune Hemolytic Anemia; Vasculitis; Amyloidosis
• Interventions: Biological: CD19/BCMA CAR T-cells

• Registration Number: NCT05263817
• Lead Sponsor: Zhejiang University

Brief Summary

A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis

Detailed Description

POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis and other diseases may only show local pathological damage or systemic lesions. If they are not diagnosed and treated in time or poorly controlled, they will progress as the course of the disease progresses. Risk of disability or even death.

Study Timeline

• Study Start: 2021-10-08
• Study First Submitted: 2021-12-01
• Status Verified: 2022-02
• Study First Submitted QC: 2022-02-21
• Last Update Submitted: 2022-02-21
• Study First Posted: 2022-03-03
• Last Update Posted: 2022-03-03
• Primary Completion: 2024-10-01
• Study Completion: 2024-10-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

• 1. Diagnosed with POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis, and the curative effect of conventional hormones, radiotherapy and chemotherapy, protease inhibitors is not good and (or) no effective treatment means.

  2. After glucocorticoids, cyclophosphamide or methotrexate treatments there are still relapsed and refractory diseases, or clearly show intolerance/toxicity to these drugs.

  3. Estimated survival time> 12 weeks; 4. Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up; 5. Patients or their legal guardians volunteer to participate in the study and sign the informed consent.

Exclusion Criteria

• Subjects with any of the following exclusion criteria were not eligible for this trial:

  1. History of craniocerebral trauma, conscious disturbance, epilepsy,cerebrovascular ischemia, and cerebrovascular, hemorrhagic diseases;
  2. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
  3. Pregnant (or lactating) women;
  4. Patients with severe active infections (excluding simple urinary tract infection and bacterial pharyngitis);
  5. Active infection of hepatitis B virus or hepatitis C virus;
  6. Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
  7. Those who have used any gene therapy products before.
  8. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
  9. Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0mg/dl;
  10. Those who suffer from other uncontrolled diseases are not suitable to join the study;
  11. HIV infection;
  12. Any situation that the researchers believe may increase the risk of patients or interfere with the test results.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
POEMS Syndrome	CD19/BCMA CAR T-cells	-
Autoimmune Hemolytic Anemia	CD19/BCMA CAR T-cells	-
Amyloidosis	CD19/BCMA CAR T-cells	-
Vasculitis	CD19/BCMA CAR T-cells	-

Primary Outcome Measures

Name	Time	Method
Dose-limiting toxicity (DLT)	Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion	Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of treatment-emergent adverse events (TEAEs)	Up to 2 years after CD19/BCMA targeted CAR T-cells infusion	Incidence of treatment-emergent adverse events \[Safety and Tolerability\]

Secondary Outcome Measures

Name	Time	Method
Titer of auto-antibody Titer of auto-antibody titer of auto-antibody	Up to 2 years after CD19/BCMA targeted CAR T-cells infusion	In peripheral blood and bone marrow
Overall response rate (ORR)	Up to 2 years after CD19/BCMA targeted CAR T-cells infusion	Proportion of subjects with complete or partial remission
Duration of remission, DOR	2 years post CD19/BCMA CAR-T cells infusion	The time from the first assessment of remission or partial remission of the disease to the first assessment of disease progression or death from any cause
Best overall response, BOR	At ≤3 month	Assessment of ORR at ≤3 month
Overall survival (OS)	From CD19/BCMA CAR-T infusion to death，up to 2 years	The time from the cell reinfusion to death due to any cause

Trial Locations

Locations (1)

The first affiliated hospital of medical college of zhejiang university; 🇨🇳 Hangzhou, Zhejiang, China