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

Amyloidosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05263817
• Other study ID #: CD19/BCMA-005
• Status: Recruiting
• Phase: Early Phase 1
• Start date: October 8, 2021
• Est. completion date: October 1, 2024

Study information

• Verified date: February 2022
• Source: Zhejiang University
• Contact: He Huang, PhD
• Phone: +8613605714822
• Email: hehuangyu[@]126.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 75
• Est. completion date: October 1, 2024
• Est. primary completion date: October 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyloidosis
• Anemia
• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Autoimmune Hemolytic Anemia
• Hemolysis
• POEMS Syndrome
• Syndrome
• Vasculitis

Intervention

Biological:
• CD19/BCMA CAR T-cells
Each subject receive CD19/BCMA CAR T-cells by intravenous infusion

Locations

Country	Name	City	State
China	The first affiliated hospital of medical college of zhejiang university	Hangzhou	Zhejiang

Sponsors (2)

Lead Sponsor	Collaborator
Zhejiang University	Yake Biotechnology Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Dose-limiting toxicity (DLT)	Adverse events assessed according to NCI-CTCAE v5.0 criteria	Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion
Primary	Incidence of treatment-emergent adverse events (TEAEs)	Incidence of treatment-emergent adverse events [Safety and Tolerability]	Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Secondary	Titer of auto-antibody Titer of auto-antibody titer of auto-antibody	In peripheral blood and bone marrow	Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Secondary	Overall response rate (ORR)	Proportion of subjects with complete or partial remission	Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Secondary	Best overall response, BOR	Assessment of ORR at =3 month	At =3 month
Secondary	Overall survival (OS)	The time from the cell reinfusion to death due to any cause	From CD19/BCMA CAR-T infusion to death,up to 2 years
Secondary	Duration of remission, DOR	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	2 years post CD19/BCMA CAR-T cells infusion