A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease

Phase 1

Recruiting

• Conditions: Antineutrophilic Cytoplasmic Antibody (ANCA)- Associated Vasculitis (AAV); Idiopathic Inflammatory Myositis (IIM); Systemic Sclerosis (SSc); Systemic Lupus Erythematosus (SLE)
• Interventions: Drug: FT819; Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Bendamustine

• Registration Number: NCT06308978
• Lead Sponsor: Fate Therapeutics

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), 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-03-06
• Study First Submitted QC: 2024-03-06
• Study First Posted: 2024-03-13
• Study Start: 2024-03-28
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-08
• Last Update Posted: 2025-04-10
• Primary Completion: 2027-09-30
• Study Completion: 2042-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 244

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).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Regimen A (Single dose with AMP)	FT819	-
Regimen A (Single dose with AMP)	Fludarabine	-
Regimen A (Single dose with AMP)	Cyclophosphamide	-
Regimen C (Two-dose with AMP)	Fludarabine	-
Regimen C (Two-dose with AMP)	Cyclophosphamide	-
Regimen B1 (Single-dose without AMP, background therapy temporarily suspended)	FT819	-
Regimen A (Single dose with AMP)	Bendamustine	-
Regimen B (Single-dose without AMP, with background therapy)	FT819	-
Regimen C (Two-dose with AMP)	FT819	-
Regimen C (Two-dose with AMP)	Bendamustine	-
Regimen D (Two-dose without AMP, with background therapy)	FT819	-

Primary Outcome Measures

Name	Time	Method
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.
Recommend Phase 2 dose (RP2D) of FT819	Up to approximately 2 years	The RP2D will be determined.

Secondary Outcome Measures

Name	Time	Method
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).

Trial Locations

Locations (4)

University of California Irvine; 🇺🇸 Irvine, California, United States

Children's Hospital Los Angeles Division Of Rheumatology; 🇺🇸 Los Angeles, California, United States

University of Minnesota Medical School; 🇺🇸 Minneapolis, Minnesota, United States

University of Nebraska Medical Center; 🇺🇸 Omaha, Nebraska, United States