HiSCs in the Treatment of Rheumatoid Arthritis

NCT06947746 | Recruiting DMC

• 1 other identifier: HWHY-hiSC-01
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This trial is a single-center, single-arm exploratory clinical study aimed at assessing the safety, tolerability, and preliminary efficacy of a single intra-articular injection of hiSCs for the treatment of rheumatoid arthritis.

Conditions

Rheumatoid Arthritis (RA)

Keywords

Sertoli cells; Rheumatoid Arthritis (RA); intra-articular injection; hiSCs

Outcome Measures

Primary Outcomes (4)

• TEAEs and SAEs

  The incidence of TEAEs and SAEs related to the study intervention

  Within 12 weeks after the injection of hiSCs

• WOMAC

  The change from baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) score (range: 0-240 cm; including pain, stiffness, and physical function subscales) for the target knee, as assessed by the subject, with higher scores indicating greater symptom severity and worse physical function

  At 12 weeks after the injection of hiSCs

• GSUS

  The change from baseline in Gray-scale ultrasound (GSUS) (range: 0-12; including suprapatellar, medial, lateral, and posterior planes) for the target knee, as assessed by musculoskeletal ultrasound, with higher scores indicating more severe synovitis

  At 12 weeks after the injection of hiSCs

• PDUS

  The change from baseline in Power Doppler Ultrasound Synovitis (PDUS) score (range: 0-15; including suprapatellar, infrapatellar, medial, lateral, and posterior compartments) for the target knee, as assessed by power Doppler ultrasound, with higher scores indicating more severe synovitis

  At 12 weeks after the injection of hiSCs

Secondary Outcomes (26)

• ACR20, ACR50, ACR70

  At Weeks 2, 4, 8, 12, and 24

• TJC

  At Weeks 2, 4, 8, 12, and 24

• SJC

  At Weeks 2, 4, 8, 12, and 24

• HAQ-DI

  At Weeks 2, 4, 8, 12, and 24

• Patient-reported pain intensity (VAS）

  At Weeks 2, 4, 8, 12, and 24

Other Outcomes (5)

• Cytomorphology

  At 12 weeks after the injection of hiSCs

• Total protein

  At 12 weeks after the injection of hiSCs

• Cytokine response

  At 12 weeks after the injection of hiSCs

Study Arms (1)

hiSCs treatment

EXPERIMENTAL

hiSCs，human induced Sertoli-like cells

Biological: hiSCs

Interventions

hiSCs BIOLOGICAL

hiSCs are derived from human induced pluripotent stem cells and exhibit immunomodulatory properties.

hiSCs treatment

Eligibility Criteria

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

You may qualify if:

• Voluntarily sign the informed consent;
• Male or female aged 18-65 years (inclusive) at the time of signing the informed consent;
• Diagnosis of RA for ≥3 months according to the ACR/EULAR 2010 Rheumatoid Arthritis Classification Criteria at the screening visit;
• At the screening visit, presence of recurrent swelling and pain in at least one knee, with a WOMAC pain score ≥4, synovial inflammation confirmed by joint ultrasound, and no significant improvement following 3 months of anti-RA treatment (including prior use of MTX standard therapy, biologics, or small molecule targeted drugs);
• Subjects must have received csDMARD therapy for ≥3 months, with a stable dose for ≥4 weeks prior to screening;
• Background treatment with stable-dose MTX standard therapy, biologics, or small molecule targeted drugs, either alone or in combination, is permitted;
• The following csDMARDs, either alone or in combination, are permitted as background treatment, provided the dose has been stable for ≥4 weeks prior to screening: oral or IV MTX (10-25 mg/week; for subjects intolerant to doses ≥10 mg/week, the dose should be ≥7.5 mg/week), SAS (≤3 g/day), hydroxychloroquine (≤400 mg/day), and LEF (≤20 mg/day);
• Stable-dose NSAIDs are permitted, provided the dose has remained stable for ≥2 weeks prior to screening;
• All females of childbearing potential must have a negative blood pregnancy test within 7 days prior to treatment initiation and must not be breastfeeding. Females not of childbearing potential may be exempt from the pregnancy test and contraception. All enrolled patients (regardless of gender) must use at least one highly effective method of contraception, including adequate barrier methods, throughout the study duration;
• Subjects must be in good overall health, able to ambulate independently (excluding those requiring a wheelchair, walker, or crutches);
• Willingness and ability to adhere to scheduled visits, treatment regimens, laboratory tests, and other study-related procedures.

You may not qualify if:

• Presence of other immune-mediated disorders at the baseline visit that may interfere with the administration or efficacy evaluation of the study intervention;
• History or current evidence of clinically significant cardiovascular, neuropsychiatric, renal, hepatic, immune, or endocrine disorders (including uncontrolled diabetes or thyroid disease), abnormal laboratory findings, or conditions requiring medications prohibited by the study protocol. "Clinically significant" refers to conditions that, in the investigator's judgment, may jeopardize subject safety or impact efficacy or safety analyses if the disease/condition exacerbates during the study;
• Subjects with positive test results for human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), or syphilis (refer to laboratory tests for details);
• Evidence of active tuberculosis (TB) or a history of active TB without adequate documented treatment;
• Any other acute or chronic disorder leading to coagulation dysfunction that, in the investigator's judgment, may compromise patient safety and/or interfere with the evaluation of target knee outcomes;
• Clinically significant infection within 1 month prior to the screening visit (requiring hospitalization and parenteral administration of antibiotics, antivirals, antifungals, etc., for ≥3 days) or active infection being treated during the screening period;
• Infection in the target knee within 3 months before baseline;
• Intra-articular corticosteroid or other drug injections in the target knee within 3 months before baseline;
• History of knee injury or prior knee surgery in the target knee within 1 year prior to the baseline visit;
• Serum transaminase (ALT or AST) levels ≥2 times the upper limit of normal (ULN) during screening;
• Creatinine clearance (Ccr) \<45 mL/min (based on the Cockcroft-Gault formula) during screening;
• Evidence of hematopoietic dysfunction during screening:
• Hemoglobin level \<9.0 g/dL or hematocrit \<30%;
• White blood cell count \<3.0×10⁹/L or absolute neutrophil count (ANC) \<1.2×10⁹/L;
• Platelet count \<100×10⁹/L;

Sponsors & Collaborators

• Shanghai Changzheng Hospital: lead

Study Sites (1)

Shanghai ChangZheng hospital

Shanghai, 200003, China

RECRUITING

MeSH Terms

Conditions

Arthritis, Rheumatoid

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Huji Xu, Ph.D, MD

  Shanghai Changzheng Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Huji Xu, Ph.D, MD

CONTACT

86021-81885514; huji_xu@tsinghua.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Chief Physician, Head of the Department of Rheumatology and Immunology, Shanghai Changzheng Hospital.

Study Record Dates

• First Submitted: March 26, 2025
• First Posted: April 27, 2025
• Study Start: May 1, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): April 1, 2027
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)