IgG4 Related Disease
Conditions
Keywords
IgG4 Related Disease, cell therapy, IgG4-RD, ACE1831
Brief summary
ACE1831 is an off-the-shelf, allogeneic gamma delta T (gdT) cell therapy derived from healthy donors, that is under investigation for the treatment in subjects with Immunoglobulin G4 Related Disease (IgG4-RD)
Detailed description
ACE1831-201 study is an Open Label, Multicenter, Single Arm Study to Assess Safety, Efficacy and Persistence of ACE1831, in Subjects with Immunoglobulin G4-Related Disease
Interventions
DRUGACE1831
ACE1831 is allogeneic gamma delta T (gdT) cell therapy. Subjects will receive ACE1831 dose based on the assigned dose escalation cohort.
Subjects assigned to receive lymphodepleting preconditioning (LDC) will receive chemotherapy cyclophosphamide ahead of ACE1831 administration.
Sponsors
Acepodia Biotech, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
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 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.
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability of ACE1831 as Assessed by Adverse Events, Clinical Laboratory Tests, Physical Examinations, ECGs, and Vital Signs | up to 72 weeks post last-ACE1831 dose | Primary Outcome Measures: Safety and Tolerability of ACE1831. Adverse Events: Incidence of TEAEs, SAEs, AESIs, and DLTs. Clinical Laboratory Abnormalities: Number of subjects with clinically significant abnormalities in protocol-defined clinical laboratory assessments compared with baseline. Physical Examination Abnormalities: Number of subjects with clinically significant changes from baseline. ECG Abnormalities: Number of subjects with clinically significant ECG changes (PR, QRS, QT/QTcF, heart rate) from baseline. Vital Signs Abnormalities: Number of subjects with clinically significant changes from baseline. Number of Subjects With Clinically Significant Changes in Vital Signs From Baseline. Number of subjects with clinically significant changes in vital signs, including temperature, respiratory rate, heart rate, blood pressure, and oxygen saturation (SpO₂), compared with baseline. For all of the above, Unit of Measure: Number of Subjects |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To assess the efficacy of ACE1831 (primary efficacy) | 24 weeks after last dose of ACE1831 | • Proportion of subjects in complete remission 24 weeks after last dose of ACE1831 |
| 3.1 To assess the efficacy of ACE1831 (secondary efficacy): Proportion of subjects who experience sustained complete remission | up to 72 weeks post-last ACE1831 dose | Proportion of subjects who experience sustained complete remission 72 weeks after last dose of ACE1831 |
| 3.2 To assess the efficacy of ACE1831 (secondary efficacy): Time to first flare | up to 72 weeks post-last ACE1831 dose | Time (days) from first dose of ACE1831 to first flare |
| 3.3 To assess the efficacy of ACE1831 (secondary efficacy): Cumulative GC usage | up to 72 weeks post-last ACE1831 dose | Cumulative GC usage (milligrams) at 24 weeks after the last dose of ACE1831 |
| 3.4 To assess the efficacy of ACE1831 (secondary efficacy): Changes in SF-12 | up to 72 weeks post-last ACE1831 dose | Changes in Quality of Life Questionnaire (QOL) Short Form 12 (SF-12) total score |
| 3.5 To assess the efficacy of ACE1831 (secondary efficacy): Changes in PGA | up to 72 weeks post-last ACE1831 dose | Changes in Physician's Global Assessment (PGA) of disease activity score (Visual Activity Score, scale range 0 -100) |
| 3.6 To assess the efficacy of ACE1831 (secondary efficacy): Time to PGA = 0 | up to 72 weeks post last-ACE1831 dose | Time (days) to PGA assessment score of 0 |
| 3.7 To assess the efficacy of ACE1831 (secondary efficacy): Changes in SGA | up to 72 weeks post last-ACE1831 dose | Changes in Subject's Global Assessment (SGA) of disease activity score (Visual Activity Score, scale range 0 - 100) |
| 3.8 To assess the efficacy of ACE1831 (secondary efficacy): Changes in SSI | up to 72 weeks post last-ACE1831 dose | Changes in IgG4-RD symptom severity index (IgG4-RD-SSI) score |
| 3.9 To assess the efficacy of ACE1831 (secondary efficacy): Changes in IgG4-RD RI | up to 72 weeks post last-ACE1831 dose | Changes in IgG4-RD Responder Index (IgG4-RD RI) score |
Countries
Japan, United States
Contacts
CONTACTAcepodia Clinical Team
Outcome results
None listed