Antineutrophilic Cytoplasmic Antibody (ANCA)- Associated Vasculitis (AAV), Idiopathic Inflammatory Myositis (IIM), Lupus Nephritis, Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc)
Conditions
Keywords
FT819, Fate Therapeutics, Idiopathic inflammatory myositis (IIM), Systemic lupus erythematosus (SLE), Systemic sclerosis (SSc), Antineutrophilic cytoplasmic antibody (ANCA)-associated vasculitis (AAV), Allogeneic CAR-T, CD19-Targeted Therapy, Cell Therapy for Autoimmune Diseases, B-Cell Depletion in Autoimmune Disease, Phase 1 Clinical Trial, Allogeneic CAR cells, Autoimmune Diseases, A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Diseases, Lupus Nephritis
Brief summary
This is a phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and anti-B-cell activity of FT819 following treatment with or without auxiliary medicinal product (AMP) in participants with moderate-to-severe active systemic lupus erythematosus (SLE) with or without nephritis, antineutrophilic cytoplasmic antibody (ANCA)-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The study will consist of a dose-escalation stage, followed by an expansion stage to further evaluate the safety and activity of FT819.
Interventions
DRUGFT819
FT819 will be administered as intravenous (IV) infusion at planned dose levels.
DRUGFludarabine
Fludarabine will be administered as an IV infusion at planned dose levels.
DRUGCyclophosphamide
Cyclophosphamide will be administered as an IV infusion at planned dose levels.
DRUGBendamustine
Bendamustine will be administered as an IV infusion at planned dose levels.
Sponsors
Fate Therapeutics
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Age: 12 to 70 years old. * Diagnosis: Must have active B-cell mediated autoimmune disease (SLE, AAV, IIM, or SSc) confirmed by standard criteria. * Disease Severity: Moderate to severe, requiring at least two prior treatments that were ineffective. * Health Status: Adequate organ function to tolerate treatment. * Consent: Able to provide informed consent or assent/obtain parental consent and comply with study procedures. Key
Exclusion criteria
* Pregnancy/Breastfeeding: Women must not be pregnant or nursing. * Severe Organ Dysfunction: Significant heart, lung, liver, or kidney impairment. * Active Infections: No recent or ongoing serious infections. * Recent Cancer or Prior Cell Therapy: No active/recent malignancies, prior CAR T-cell therapy, or organ transplant. * Allergies: No known allergies to study treatments. * Weight Restriction: Must weigh at least 50 kg (110 lbs).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-emergent adverse events (TEAEs) | Up to approximately 2 years | The number of participants with TEAEs will be reported. |
| Number of participants with serious TEAEs | Up to approximately 2 years | The number of participants with serious TEAEs will be reported. |
| Number of participants with dose-limiting toxicities (DLTs) | Up to approximately 29 days | The number of participants with DLTs will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma concentration of FT819 | At designated time points up to approximately 29 days | The plasma concentration of FT819 will be determined. |
| Impact of treatment on quality of life | Up to approximately 2 years | Assess changes in patient-reported health outcomes using the SF-36 survey. |
| Disease Activity | Up to approximately 2 years | Evaluate improvements in disease-specific measures, such as: * SLE: Reduction in SLEDAI-2K score, rates of achieving DORIS, LLDAS * AAV: Proportion of patients achieving remission (BVAS v3) and relapse-free survival. * IIM: Proportion of patients achieving Myositis Response Criteria Total Improvement Score (MRC TIS major, moderate, minimal). * SSc: Changes in modified Rodnan Skin Score (mRSS) and lung function tests (FVC, DLCO). |
| Disease Activity in Participants with Lupus Nephritis | Up to approximately 2 years | Change from baseline in urine protein to creatinine ratio (UPCR) |
Countries
France, Sweden, United Kingdom, United States
Contacts
CONTACTFate Clinical Trials
Outcome results
None listed