CAR-T Cells Targeting Autoimmune Diseases

NCT ID: NCT05459870

Last Updated: 2022-07-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-31

Study Completion Date

2026-06-30

Brief Summary

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The purpose of this study is to assess the feasibility, safety and efficacy of CAR-T cell therapy in patients with autoimmune disease. Another goal of the study is to learn more about the safety and function of the CAR-T cells and their persistency in autoimmune disease patients.

Detailed Description

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Autoimmune disease refers to the disease in which the immune system reacts to the host's own body and causes damage to tissues and organs. At present, the pathogenesis of various autoimmune diseases is still not well understood, but an imbalanced immune tolerance plays a key role in this process.

An ideal therapy to autoimmune disease should eradicate pathogenic autoimmune cells but retain the protective immunity. The chimeric antigen receptor-modified T (CAR-T) cell technology has proven to be highly effective in targeting B cell malignancies, and the treatment-induced B cell and antibody deficiencies have implications for treating autoantibody-related autoimmune diseases. Studies have shown that CAR-T cells targeting B cell surface molecules can kill autoreactive B lymphocytes in pemphigus vulgaris (PV) and systemic lupus erythematosus (SLE) patients. Thus, CAR-T cell technology targeting B cells has potential in treating autoimmune diseases including PV, SLE, autoimmune hemolytic anemia, Sjogren's syndrome etc..

CD19-specific CAR is based on activation of intracellular signalijng domains of T cells by the extracellular single chain variable fragment (scFv) antibody against CD19. The activated CAR-T cells can target and kill B cells. The investigation plans to use genetically modified T cells to express a 4th generation lentiviral anti-CD19 CAR with an inducible caspase 9 self-withdrawal gene (4SCAR) to increase the safety of this specific approach. Besides targeting CD19, specific CARs targeting other B cell surface molecules including BCMA, CD138, and BAFF-R will also be included in the treatment regimen. Based on accumulated experiences, the 4SCAR T cells have shown high safety profile without serious cytokine release syndrome (CRS) or neural toxicities in patients. Through this trial, the safety and long term efficacy of the B cell-specific 4SCAR T cell therapy will be evaluated, providing clinical evidence supporting the application of 4SCAR-T cell technology in the treatment of autoimmune diseases.

Conditions

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Autoimmune Diseases

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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4SCAR T Cell Therapy for autoimmune diseases

Group Type EXPERIMENTAL

4SCAR T cells

Intervention Type BIOLOGICAL

Infusion of 4SCAR T cells at 10\^6 cells/kg body weight via IV

Interventions

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4SCAR T cells

Infusion of 4SCAR T cells at 10\^6 cells/kg body weight via IV

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. age older than 18 years.
2. expression of B cell surface molecules.
3. the KPS score over 80 points, and survival time is more than 3 months.
4. greater than Hgb 80 g/L.
5. no contraindications to blood cell collection.

Exclusion Criteria

1. accompanied with other active diseases and difficult to assess treatment response.
2. bacterial, fungal, or viral infection, unable to control.
3. living with HIV.
4. active HBV or HCV infection.
5. pregnant and nursing mothers.
6. under systemic steroid treatment within a week of the treatment.
7. prior failed CAR-T treatment.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Guilin Hospital of Chinese Traditional and Western Medicine

UNKNOWN

Sponsor Role collaborator

Shenzhen Geno-Immune Medical Institute

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, China

Site Status RECRUITING

Guilin Hospital of Chinese Traditional and Western Medicine

Guilin, Guangxi, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Lung-Ji Chang, PhD

Role: CONTACT

86-0755-86725195

Facility Contacts

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Lung-Ji Chang, PhD

Role: primary

86-0755-86725195

Qiyong Zhu

Role: primary

Other Identifiers

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GIMI-IRB-22010

Identifier Type: -

Identifier Source: org_study_id

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