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

Primary Purpose

POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia

Status

Recruiting

Phase

Early Phase 1

Locations

China

Study Type

Interventional

Intervention

CD19/BCMA CAR T-cells

About this trial

This is an interventional treatment trial for POEMS Syndrome focused on measuring CD19 CAR-T, BCMA CAR-T, POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

The first affiliated hospital of medical college of zhejiang universityRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Arm Label

POEMS Syndrome

Amyloidosis

Autoimmune Hemolytic Anemia

Vasculitis

Arm Description

Outcomes

Primary Outcome Measures

Dose-limiting toxicity (DLT)

Adverse events assessed according to NCI-CTCAE v5.0 criteria

Incidence of treatment-emergent adverse events (TEAEs)

Incidence of treatment-emergent adverse events [Safety and Tolerability]

Secondary Outcome Measures

Titer of auto-antibody Titer of auto-antibody titer of auto-antibody

In peripheral blood and bone marrow

Overall response rate (ORR)

Proportion of subjects with complete or partial remission

Best overall response, BOR

Assessment of ORR at ≤3 month

Overall survival (OS)

The time from the cell reinfusion to death due to any cause

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

Full Information

NCT ID

NCT05263817

First Posted

December 1, 2021

Last Updated

February 21, 2022

Sponsor

Zhejiang University

Collaborators

Yake Biotechnology Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT05263817

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2022

Overall Recruitment Status

Recruiting

Study Start Date

October 8, 2021 (Actual)

Primary Completion Date

October 1, 2024 (Anticipated)

Study Completion Date

October 1, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Zhejiang University

Collaborators

Yake Biotechnology Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis

Keywords

CD19 CAR-T, BCMA CAR-T, POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

75 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

POEMS Syndrome

Arm Type

Experimental

Arm Title

Amyloidosis

Arm Type

Experimental

Arm Title

Autoimmune Hemolytic Anemia

Arm Type

Experimental

Arm Title

Vasculitis

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

CD19/BCMA CAR T-cells

Other Intervention Name(s)

CD19/BCMA CAR T-cells injection

Intervention Description

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

Primary Outcome Measure Information:

Title

Dose-limiting toxicity (DLT)

Description

Adverse events assessed according to NCI-CTCAE v5.0 criteria

Time Frame

Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion

Title

Incidence of treatment-emergent adverse events (TEAEs)

Description

Incidence of treatment-emergent adverse events [Safety and Tolerability]

Time Frame

Up to 2 years after CD19/BCMA targeted CAR T-cells infusion

Secondary Outcome Measure Information:

Title

Titer of auto-antibody Titer of auto-antibody titer of auto-antibody

Description

In peripheral blood and bone marrow

Time Frame

Up to 2 years after CD19/BCMA targeted CAR T-cells infusion

Title

Overall response rate (ORR)

Description

Proportion of subjects with complete or partial remission

Time Frame

Up to 2 years after CD19/BCMA targeted CAR T-cells infusion

Title

Best overall response, BOR

Description

Assessment of ORR at ≤3 month

Time Frame

At ≤3 month

Title

Overall survival (OS)

Description

The time from the cell reinfusion to death due to any cause

Time Frame

From CD19/BCMA CAR-T infusion to death，up to 2 years

Title

Duration of remission, DOR

Description

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

Time Frame

2 years post CD19/BCMA CAR-T cells infusion

10. Eligibility

Sex

All

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

He Huang, PhD

Phone

+8613605714822

hehuangyu@126.com

First Name & Middle Initial & Last Name or Official Title & Degree

Yongxian Hu, PhD

Phone

+8615957162012

huyongxian2000@aliyun.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

He Huang, PhD

Organizational Affiliation

First Affiliated Hospital of Zhejiang University

Official's Role

Principal Investigator

Facility Information:

Facility Name

The first affiliated hospital of medical college of zhejiang university

City

Hangzhou

State/Province

Zhejiang

ZIP/Postal Code

310003

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

He Huang, PhD

Phone

86-13605714822

hehuangyu@126.com

First Name & Middle Initial & Last Name & Degree

Yongxian Hu, PhD

Phone

+8615957162012

huyongxian2000@aliyun.com

12. IPD Sharing Statement

Learn more about this trial

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

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