GT719 Injection for Moderate to Severe Refractory Autoimmune Diseases

Not Applicable

Not yet recruiting

• Conditions: Autoimmune Diseases
• Interventions: Biological: GT719 Injection

• Registration Number: NCT07122076
• Lead Sponsor: Grit Biotechnology

Brief Summary

This study is a prospective single-arm open-label clinical trial, aims to evaluate the safety, efficacy, and cellular pharmacokinetics of GT719 Injection in patients with moderate to severe refractory autoimmune diseases. A total of 10 subjects will be enrolled in this study.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-07-29
• Status Verified: 2025-08
• Study First Submitted QC: 2025-08-11
• Last Update Submitted: 2025-08-11
• Study First Posted: 2025-08-14
• Last Update Posted: 2025-08-14
• Study Start: 2025-08-20
• Primary Completion: 2028-08-19
• Study Completion: 2028-08-19

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

Exclusion Criteria

1. SLE participants:

   1. Drug induced SLE;
   2. Patients with lupus crisis or concomitant diseases who require the use of contraindicated drugs according to the protocol are deemed unsuitable for inclusion by the investigator.

2. IIM participants

   1. Documented inclusion body myositis (IBM), drug-induced PM or DM, tumor associated PM or DM, non-inflammatory myopathy (such as muscular dystrophy);
   2. Uncontrolled extramuscular disease damage related to PM or DM:

   ILD: FVC<55% or requiring oxygen therapy;

   Severe swallowing difficulties, as determined by investigator, increase the risk of patients participating in clinical trials;

   Severe cardiac manifestations (such as congestive heart failure, arrhythmia, conduction abnormalities requiring treatment, or myocardial infarction) have been determined by investigator to increase the risk of patients participating in clinical trials.

3. SSc participants

   1. Moderate to severe pulmonary arterial hypertension (PAH) associated with SSc that cannot be controlled by drug therapy;
   2. Rapid progressive SSc related low gastrointestinal (small and large intestine) involvement (requiring parenteral nutrition); Active dilation of gastric antral blood vessels;
   3. Uncontrolled or rapidly progressing ILD with oxygen saturation (SaO2) <92% (in still indoor air); Or require mechanical respiratory assistance (ventilator) within one year prior to signing the informed consent form.

4. Has a history of severe hypersensitivity reactions or allergies;

5. Contraindications or hypersensitivity reactions to any components of fludarabine, cyclophosphamide, and experimental drugs;

6. Suffering from the following heart diseases:

   1. NYHA Grade III or IV congestive heart failure;
   2. Has experienced a myocardial infarction or undergone coronary artery bypass surgery within the 6 months prior to the screening period;
   3. A clinically significant history of ventricular arrhythmia or unexplained syncope, not caused by vasovagal nerve response or dehydration, or a corrected QT interval (QTc)>480 ms during screening;
   4. History of severe non ischemic cardiomyopathy;

7. Any active malignant tumors or history of malignant tumors within the past 5 years before screening. Excluding the following situations: early-stage tumors that have received curative treatment (in situ or stage I tumors, non ulcerative primary melanoma with a depth of<1 mm and no involvement of lymph nodes), basal cell carcinoma of the skin, squamous cell carcinoma of the skin, cervical in situ cancer, or breast in situ cancer that has received potential curative treatment;

8. Individuals with clinically significant bleeding symptoms or clear bleeding tendencies within the 6 months prior to screening, such as gastrointestinal bleeding, hemorrhagic gastric ulcers, etc; Hereditary or acquired bleeding and thrombophilia tendencies (such as hemophilia, coagulation dysfunction, splenic hyperfunction, etc.); Occurrence of arteriovenous thrombosis events within 6 months prior to screening, such as cerebrovascular disease (including cerebral hemorrhage, cerebral infarction, etc.), deep vein thrombosis, and/or pulmonary embolism;

9. When screening, there may be serious underlying medical conditions, such as:

   1. There is evidence to suggest the presence of uncontrollable viruses, bacteria, fungi, or other infections that require systemic intravenous treatment;
   2. There is clear clinical evidence indicating the presence of dementia or changes in mental state;
   3. Any other history of central nervous system disorders or neurodegenerative diseases, such as epilepsy, seizures, paralysis, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, mental illness;

10. Any of the following test results is positive:

    1. Positive for human immunodeficiency virus (HIV) antibodies;
    2. Hepatitis B surface antigen (HBsAg) positive; Or if the hepatitis B core antibody (HBcAb) is positive and the hepatitis B virus (HBV) - DNA is higher than the detection limit of the analytical method;
    3. Hepatitis C virus (HCV) antibody positive and HCV RNA above the detection limit of the analytical method;
    4. Syphilis antibody positive (false positive result due to reasons other than the disease);

11. Cytomegalovirus (CMV) (DNA) and Epstein Barr virus (EBV) (DNA) tests positive;

12. Active tuberculosis or latent tuberculosis without proper treatment before screening;

13. Received other clinical trial drugs within 4 weeks prior to the signing of the informed consent form (ICF), or the ICF signing date is within 5 half lives of the drug from the last use of the drug in the previous clinical trial (whichever is longer);

14. Received plasma exchange therapy or immunoadsorption therapy within 4 weeks prior to lymphodepleting chemotherapy;

15. Used drugs targeting B cells, including but not limited to rituximab, belimumab, tacrolizumab, etc., within one week before lymphodepleting chemotherapy;

16. Used tacrolimus, cyclosporine, azathioprine, mycophenolate mofetil, mycophenolate mofetil, methotrexate, etc. within 2 weeks before lymphodepleting chemotherapy;

17. Used neonatal Fc receptor (FcRn) antagonist therapy (such as Efgartigimod) within 3 weeks before lymphodepleting chemotherapy;

18. Within 3 weeks prior to lymphodepleting chemotherapy, complement inhibition therapy (such as Ecuzumab) has been used;

19. Received attenuated live vaccine within 4 weeks before lymphodepleting chemotherapy;

20. Having undergone major surgery within the 8 weeks prior to screening, or planning to undergo surgery during the study period;

21. Medical history of organ transplantation;

22. Previously received CAR-T product therapy targeting any target (excluding GT719 therapy);

23. According to the investigator's judgment, the situations that hinder participants from participating in the entire trial, confound the trial results, or participate in the trial that are not in the best interests of the participants.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
GT719 Injection treatment group	GT719 Injection	GT719 Injection

Primary Outcome Measures

Name	Time	Method
Proportion of participants experiencing dose limiting toxicity	28 days	Proportion of participants experiencing dose limiting toxicity (DLT) within 28 days after cell infusion
Incidence of treatment-emergent adverse events	3 months	Safety assessments are conducted using the NCI-CTCAE version 5.0 standards

Secondary Outcome Measures

Name	Time	Method
Efficacy outcomes for SLE	14 days,1, 2, 3 and 6 Months post GT719 infusion	SLE Response index 4(SR-4) response: Min/Max Value: Not specified; a decrease in score indicates improvment, higher scores indicate worse outcome
Efficacy outcomes for Systemic Sclerosis	14 days,1, 2, 3 and 6 Months post GT719 infusion	ACR-CRISS score (CRISS score≥0.6 improvment, \<0.6 no improvement)
Efficacy outcomes for Inflammatory Myopathy	14 days,1, 2, 3 and 6 Months post GT719 infusion	Total lmprovement Score (TlS): Min/Max Value: Not specified; an increase in score indicates improvement, higher scores indicate better outcome.

Trial Locations

Locations (1)

Shanghai Changzheng Hospital; 🇨🇳 Shanghai, China