Clinical Study of CD19 CAR-T in the Treatment of Refractory Systemic Sclerosis

NCT06792344 | Recruiting Phase 1 DMC

• 1 other identifier: 2024-IRB-0306-P-01
• Study Type: interventional
• Target: 12
• Locations: 2 countries
• Sites: 2

Brief Summary

This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of childhood-onset systemic sclerosis

Conditions

Systemic Sclerosis (SSc)

Outcome Measures

Primary Outcomes (1)

• Efficacy endpoint

  the Change in the modified Rodnan Skin Score (mRSS) from baseline at 6 months

  6 months

Secondary Outcomes (2)

• efficiency endpoint

  the first month ,the second month ,the sixth month

• PK parameter

  28 days and 90 days

Study Arms (1)

anti-CD19 CAR-T cells in the treatment of SSc

EXPERIMENTAL

Other: anti-CD19 CAR-T cells; Drug: anti-CD19 CAR-T cells

Interventions

anti-CD19 CAR-T cells OTHER

Intravenous injection the targeting CD19 CAR T cell

anti-CD19 CAR-T cells in the treatment of SSc

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Gender unlimited, age ≥5 years;
• Meet the classification criteria for Systemic sclerosis (SSc) as defined by the 2013ACR/EULAR standards;
• any one of the anti-nuclear antibodies or systemic sclerosis specific antibodies positive;
• Improved Rodnan skin score (mRSS) ≥15 points (total 51 points);
• Major organ functions were basically normal: left ventricular ejection fraction (LVEF) ≥55%, no obvious abnormality in electrocardiogram; eGFR≥30ML/min/1.73m2；AST and ALT≤3.0ULN, total bilirubin ≤2.0×ULN; SpO2≥92%, DLCO≥ 40% of predicted value, FVC≥ 50% of predicted value;
• The subject of childbearing age has a negative urine pregnancy test result and agrees to take effective contraceptive measures during the test period until 1 year after the infusion;
• The patient or his/her guardian agrees to participate in the clinical trial and signs an informed consent indicating that he/she understands the purpose and procedure of the clinical trial and is willing to participate in the study

You may not qualify if:

• （1） Severe pulmonary hypertension (PHA) (mean pulmonary pressure \> 45mmHg) or other severe major organ involvement; (2) have previously received CAR T cell therapy (except those whose safety risks have been ruled out by the investigators); (3) patients with active central nervous system diseases, such as epilepsy, cerebrovascular ischemia/hemorrhage, dementia, cerebellar diseases, etc.; (4) Congenital heart disease or history of acute myocardial infarction within 6 months before screening, or severe arrhythmias (including multi-source frequent supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with a large number of pericardial effusion, serious myocarditis, etc.; Or patients with unstable vital signs who require vasoactive drugs to maintain blood pressure; (5) Active tuberculosis at the time of screening; (6) There are active infections or uncontrollable infections that require systemic treatment at the time of screening; (7) Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; Acute graft-versus-host disease (GVHD) of grade 2 or above was present within 2 weeks prior to screening; (8) Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA detection greater than the normal range; Hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Positive for human immunodeficiency virus (HIV) antibodies; Treponema pallidum antibody positive; (9) Had received live vaccine within 4 weeks before screening; (10) Positive blood pregnancy test; (11) Patients with known malignant diseases such as tumors before screening; (12) Patients who had participated in other interventional clinical trials within 3 months before enrollment; (13) Situations in which other investigators consider it inappropriate to participate in the study

Sponsors & Collaborators

• The Children's Hospital of Zhejiang University School of Medicine: lead
• Guangzhou Women and Children's Medical Center: collaborator

Study Sites (2)

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 0571, China

RECRUITING

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310053, Christmas Island

RECRUITING

MeSH Terms

Conditions

Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Central Study Contacts

Jianhua Mao Children's Hospital, Zhejiang University School of Medicine, PHD

CONTACT

13516819071; maojh88@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: professor

Model Details: CD19 CAR-T

Study Record Dates

• First Submitted: December 23, 2024
• First Posted: January 24, 2025
• Study Start: December 15, 2024
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2027
• Last Updated: December 22, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1); CN (1)