臨床研究等提出・公開システム

Phase 1b/2a Prospective, Open Label, Multicenter, Single Arm Study to Assess Safety, Efficacy and Persistence of ACE1831, in Subjects with Immunoglobulin G4-Related Disease

Phase 1b/2a Prospective, Open Label, Multicenter, Single Arm Study to Assess Safety, Efficacy and Persistence of ACE1831, in Subjects with Immunoglobulin G4-Related Disease

Nakanishi Shinya

Syneos Health Japan K.K.

10F Umeda Daibiru, 3-3-10 Umeda, Kita-ku, Osaka

ACE1831@syneoshealth.com

Nakanishi Shinya

Syneos Health Japan K.K.

10F Umeda Daibiru, 3-3-10 Umeda, Kita-ku, Osaka

+81-80-4098-7100

ACE1831@syneoshealth.com

Pending

Jan. 30, 2026

Interventional

single arm study

open(masking not used)

dose comparison control

single assignment

treatment purpose

To be eligible for this study, all of the following inclusion criteria must be met:
- Signed Informed Consent
- Male or female >= 18 to 75 years of age
- Active IgG4-RD flare at screening with IgG4-RD Responder Index at least 2, confirmed by symptoms, labs, and/or imaging.
- History of IgG4-RD involving at least 2 organs/sites, and current flare involves at least 1 organ/site (excluding lymph nodes) requiring treatment.
- Elevated serum IgG4 above the upper limit of normal at screening.
- Able to receive glucocorticoids for current flare and taper to 0 mg by Day -5.
- Contraception agreement per protocol from screening through 24 weeks after last ACE1831 dose [no lymphodepletion conditionin (LDC)] or 12 months after last LDC dose (with LDC)
- For sites in China only: prior treatment failure to glucocorticoids and at least one immunosuppressive agent.

An individual who meets any of the following criteria will be excluded from participation in this trial.
- Significant conditions that impair ability to receive study treatment or comply.
- Predominant fibrosis in affected organs.
- Active/latent infection that would interfere with therapy(including HBV, HCV, HIV, TB, syphilis) or significant recent infection per protocol.
- Known immunodeficiency state.
- NYHA class III/IV heart disease.
- Severe allergy/hypersensitivity to monoclonal antibodies or relevant study agents.
- Malignancy within 5 years (protocol exceptions apply).
- Recent investigational agent exposure.
- Recent B-cell depleting therapy (anti-CD20/anti-CD19) unless reconstitution per protocol.
- Live/attenuated vaccine within 2 months.
- Pregnant or breastfeeding.
- Inadequate organ function/blood counts per protocol.

Both

Immunoglobulin G4-Related Disease

The single, open label study arm includes 3 dose escalation cohorts:

Cohort 1:
Cohort 1a: Receives ACE1831 (Dose Level 1) with LDC.
Cohort 1b: Receives ACE1831 (Dose Level 1) without LDC.
Cohort 2: Receives ACE1831 (Dose Level 2) with or without LDC depending on assignment.
Cohort 3: Receives ACE1831 (Dose Level 3) with or without LDC depending on assignment.

Drug: ACE1831
ACE1831 is allogeneic gamma delta T (gdT) cell therapy. Subjects will receive ACE1831 dose based on the assigned dose escalation cohort.

Drug: Lymphodepleting chemotherapy
Subjects assigned to receive lymphodepleting preconditioning (LDC) will receive chemotherapy cyclophosphamide ahead of ACE1831 administration.

To assess the safety and tolerability of ACE1831 in subjects with IgG4-RD
- To assess the incidence of Adverse Events (AEs), [AEs including Treatment Emergent AEs, Serious AEs (SAEs), AEs of Special Interests (AESIs), and dose limiting toxicities (DLTs)] (unit: number of AEs).
- To assess number of subjects with clinically significant changes in clinical laboratory tests from baseline (unit: number of subjects).
- To assess number of subjects with clinically significant changes in physical examination results from baseline (unit: number of subjects).
- To assess number of subjects with clinically significant changes in electrocardiograms (ECGs) results (combined assessment of PR, QRS, QT, and QTcF interval, and heart rate) from baseline (unit: number of subjects).
- To assess number of subjects with clinically significant changes in vital signs (temperature, respiratory rate, heart rate, blood pressure, heart rate, and SpO2 ) from baseline (unit: number of subjects).

To assess the efficacy of ACE1831
- Proportion of subjects in complete remission 24 weeks after last dose of ACE1831 (primary efficacy)
- Proportion of subjects who experience sustained complete remission 72 weeks after last dose of ACE1831 (secondary efficacy)
- Time (days) from first dose of ACE1831 to first flare (secondary efficacy)
- Cumulative GC usage (milligrams) at 24 weeks after the last dose of ACE1831 (secondary efficacy)
- Changes in Quality of Life Questionnaire (QOL) Short Form 12 (SF-12) total score (secondary efficacy)
- Changes in Physician Global Assessment (PGA) of disease activity score (Visual Activity Score, scale range 0 -100) (secondary efficacy)
- Time (days) to PGA assessment score of 0 (secondary efficacy)
- Changes in Subject Global Assessment (SGA) of disease activity score (Visual Activity Score (secondary efficacy)
- Changes in IgG4-RD symptom severity index (IgG4-RD-SSI) score (secondary efficacy)
- Changes in IgG4-RD Responder Index (IgG4-RD RI) score (secondary efficacy)

+81-76-218-8347

Nov. 27, 2025

No

NA

US/China