A Clinical Study to Evaluate the Safety and Efficacy of GT801 Injection in the Treatment of Moderate-to-Severe Refractory Autoimmune Diseases

NCT07333118 | Recruiting Early Phase 1

• 1 other identifier: GRIT-CD-CHN-801-002
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-arm, open-label, dose-escalation and dose-expansion clinical trial designed to evaluate the safety, efficacy, and cellular metabolism kinetics of GT801 in the treatment of moderate-to-severe refractory autoimmune diseases.

Conditions

Moderate to Severe Refractory Autoimmune Disease

Outcome Measures

Primary Outcomes (2)

• Proportion of participants experiencing dose limiting toxicity

  The proportion of participants with dose-limiting toxicity (DLT) occurring within 28 days after infusion

  28 days

• Adverse Events (AEs) occurring after infusion and their proportions

  Adverse Events (AEs) occurring after infusion and their proportions

  3 months

Secondary Outcomes (11)

• Efficacy outcomes for Systemic Lupus Erythematosus (SLE)

  1, 2, 3 and 6 Months post GT801 infusion

• Efficacy outcomes for Idiopathic Inflammatory Myopathies (IIM)

  1, 2, 3 and 6 Months post GT801 infusion

• Efficacy outcomes for Systemic Sclerosis (SSc)

  1, 2, 3 and 6 Months post GT801 infusion

• Efficacy Outcomes for ANCA-Associated Vasculitis (AAV)

  6 and 12 Months post GT801 infusion

• Efficacy Outcomes for Participants with Sjogren's Syndrome

  6 and 12 Months post GT801 infusion

Other Outcomes (4)

• Exploration of Peripheral Blood Lymphocyte Subsets and B Lymphocyte Subsets: Changes in T Cells and NK Cells

  1, 3, 6, 9 Months post GT801 infusion

• Exploration of Peripheral Blood Lymphocyte Subsets and B Lymphocyte Subsets: Duration of B-Cell Depletion

  3, 6, 9 Months post GT801 infusion

• Exploration and analysis of BCR repertoire diversity via BCR sequencing

  1, 3, 6, 9 Months post GT801 infusion

Study Arms (1)

GT801 Injection treatment group

EXPERIMENTAL

GT801 Injection

Biological: GT801 Injection

Interventions

GT801 Injection BIOLOGICAL

GT801 Injection

GT801 Injection treatment group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. The participant or their legal representative voluntarily signs a written informed consent form and is willing and able to comply with the study procedures.
• \. Aged 18 to 65 years old (inclusive) at the time of signing the informed consent, regardless of gender.
• \. Participants with Systemic Lupus Erythematosus (SLE) must meet the following criteria:
• a) Fulfill the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria for SLE.
• b) Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2000) score ≥ 6, with at least one British Isles Lupus Assessment Group Index (BILAG-2004) Grade A (severe manifestation) or two Grade B (moderate manifestation) organ scores, or both; or SLEDAI-2000 score ≥ 8.
• c) Meet the definition of refractory or relapsing disease: No response to conventional treatment for more than 6 months, or disease reactivation after achieving remission. Conventional treatment is defined as the use of glucocorticoids and cyclophosphamide plus at least one of the following immunomodulatory agents: antimalarials, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, and biological agents including rituximab, belimumab, and tabalumab.
• \. Participants with Idiopathic Inflammatory Myopathy (IIM) must meet the following criteria:
• a) Fulfill the 2017 EULAR/ACR classification criteria for inflammatory myopathy (including Dermatomyositis \[DM\], Polymyositis \[PM\], Antisynthetase Syndrome \[ASS\], and Necrotizing Myopathy \[NM\]).
• b) Positive for myositis-specific antibodies or myositis-associated antibodies.
• c) Must have moderate to severe IIM at screening, defined as Manual Muscle Testing-8 (MMT-8) score ≥ 142, or MMT-8 score \< 142 plus meeting 2 of the following criteria:
• \) Physician Global Assessment (PGA) (Visual Analog Scale \[VAS\]) ≥ 2 cm (on a 10-cm VAS).
• \) Patient Global Assessment (PtGA) (VAS) ≥ 2 cm (on a 10-cm VAS).
• \) Health Assessment Questionnaire (HAQ) score \> 0.25.
• \) Elevation of one or more muscle enzymes (creatine kinase \[CK\], lactate dehydrogenase \[LDH\], aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\]) ≥ 1.5 × Upper Limit of Normal (ULN).
• d) Meet the definition of refractory/relapsing or progressive disease:

You may not qualify if:

• \. Participants with SLE
• a. Drug-induced systemic lupus erythematosus.
• b. Participants with lupus crisis; or those with concurrent diseases requiring the use of protocol-prohibited drugs, who are deemed ineligible for enrollment by the investigator.
• \. Participants with IIM
• b. Uncontrolled extramuscular disease manifestations related to PM or DM:
• \) ILD: Forced vital capacity (FVC) \< 55% or requirement for oxygen therapy.
• \) Severe dysphagia manifestations that, in the investigator's judgment, would increase the participant's risk of participating in the clinical trial.
• \) Severe cardiac manifestations (e.g., congestive heart failure, cardiac arrhythmia, conduction abnormalities requiring treatment, or myocardial infarction) that, in the investigator's judgment, would increase the participant's risk of participating in the clinical trial.
• \. Participants with SSc
• a. Severe SSc-associated pulmonary arterial hypertension (PAH) that is uncontrollable with medical treatment.
• b. Rapidly progressive SSc-related lower gastrointestinal tract (small and large intestine) involvement requiring parenteral nutrition; active gastric antral vascular ectasia.
• c. Uncontrolled or rapidly progressive ILD with oxygen saturation (SaO₂) \< 92% (on room air at rest); or requirement for mechanical ventilation support within 1 year prior to signing the informed consent form.
• \. Participants with ANCA-Associated Vasculitis (AAV)
• a. Presence of rapidly progressive glomerulonephritis, acute mononeuritis multiplex, or central nervous system (CNS) involvement unrelated to AAV at screening.
• b. Life-threatening severe vasculitis (including diffuse alveolar hemorrhage, respiratory failure, intestinal perforation or massive hemorrhage, cerebral vasculitis, cardiac vasculitis, etc.).

Sponsors & Collaborators

• Grit Biotechnology: lead
• Shanghai Changzheng Hospital: collaborator

Study Sites (1)

Shanghai Changzheng Hospital

Shanghai, Shanghai Municipality, 200003, China

RECRUITING

Central Study Contacts

Huji Xu

CONTACT

+8613671609764; xuhuji@smmu.edu.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2025
• First Posted: January 12, 2026
• Study Start: November 7, 2025
• Primary Completion (Estimated): March 31, 2028
• Study Completion (Estimated): March 31, 2028
• Last Updated: January 12, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)