FT839 in Autoimmune Diseases

Phase 2RecruitingNCT07800871

Fate Therapeutics446 enrolled

Overview

The primary objectives of this trial are to evaluate the safety and tolerability and to determine the maximum tolerated dose (MTD) or recommended Phase 2 dose of FT839 with or without rituximab and/or background therapy and/or conditioning therapy.

This is a multicenter, Phase 1/2, open-label trial designed to evaluate the safety, pharmacokinetics (PK), anti-B-cell activity, and clinical activity of FT839 in participants with moderate-to-severe ANCA-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), and systemic sclerosis (SSc). Participants will receive FT839 as monotherapy or in combination with rituximab, with or without conditioning therapy and/or stable background therapy. Participants will be enrolled in 2 stages during the Phase 1 portion of the trial: a dose-escalation stage and a dose-expansion stage. In the dose-escalation stage, safety and tolerability will be assessed to define the MTD (or through the maximum assessed dose \[MAD\] in the absence of dose-limiting toxicities \[DLTs\] defining the MTD). The DLT evaluation period will extend from Day 1 through Day 29. Participants will be followed during the post-treatment follow-up period for up to 2 years after the first dose of FT839, followed by long-term-follow-up for safety and survival for up to 15 years after the first dose of FT839. In the dose-expansion stage, participants will be enrolled into disease-specific cohorts to further evaluate the safety and activity of FT839. Following completion of the Phase 1 portion, the Phase 2 portion of the trial will further evaluate the efficacy of FT839 within each disease cohort.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

446

Conditions

ANCA-Associated Vasculitis (AAV)Idiopathic Inflammatory Myositis (IIM)Rheumatoid Arthritis (RA)Systemic Lupus Erythematosus (SLE)Systemic Sclerosis (SSc)

Interventions

FT839BIOLOGICAL

Single Intravenous (IV) infusion of FT839 administered on Day 1 and Day 4

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Age ≥18 to ≤70 years * Must have active B-cell mediated autoimmune disease (AAV, IIM, RA, SLE, or SSc) confirmed by standard criteria * Moderate to severe disease, requiring at least two prior treatments that were ineffective * Adequate organ function to tolerate treatment * Able to provide informed consent and comply with study procedures Exclusion Criteria: * Diagnosis of more than 1 disease under study (AAV, IIM, RA, SSc, or SLE) or overlap syndrome * Women must not be pregnant or nursing * Severe Organ Dysfunction: Significant heart, lung, liver, or kidney impairment. * Active or chronic infections * Active or recent malignancies * Prior CAR T-cell therapy or organ transplantation * Known allergies to study treatments * Body weight \<45 kg * Active central nervous system (CNS) symptoms attributable to autoimmune disease or nonmalignant CNS disease within 12 months prior to trial intervention * Receipt of any anti-CD19- or anti-CD20-directed therapy within 6 months prior to trial intervention

Locations (1)

Providence Medical Foundation

Fullerton, California, United States

RECRUITING

Outcomes

Primary Outcomes

Phase 1: Incidence of Dose-limiting Toxicity, Adverse Events, and Serious Adverse Events

Incidence and severity of dose-limiting Toxicity (DLT)s, adverse event (AE)s, and serious adverse event (SAE)s

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: Change from baseline in Birmingham Vasculitis Activity Score

Birmingham Vasculitis Activity Score (BVAS) will be measured to evaluate the efficacy of FT839 in AAV. The BVAS score ranges from 0 to 63, with lower scores indicating better outcomes.

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: Change from baseline in Manual muscle testing-8

Manual muscle testing-8 (MMT-8) will be measured to evaluate the efficacy of FT839 in IIM. The MMT-8 score ranges from 0 to 150, with higher scores indicating better outcomes.

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: Change from baseline in Disease Activity Score using 28 joint counts and C-reactive protein (a composite measure)

Disease Activity Score using 28 joint counts and C-reactive protein (DAS28-CRP) will be measured to evaluate the efficacy of FT839 in RA. The validated composite measure DAS28-CRP score ranges from 0.0 to 9.4, with lower scores indicating better outcomes.

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: Change from baseline in SLE Disease Activity Index 2000

SLE Disease Activity Index 2000 (SLEDAI-2K) score will be measured to evaluate the efficacy of FT839 in SLE. The SLEDAI-2K score ranges from 0 to 105, with lower scores indicating better outcomes.

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: Change from baseline in Modified Rodnan skin score

Central Contacts

Fate Clinical Trials

CONTACT

858-875-1800 clinicaltrials@fatetherapeutics.com

Natalie Shiff, MD

CONTACT

Data from ClinicalTrials.gov

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Secondary Outcomes

Phase 1: Maximum concentration and area under the curve of FT839 in peripheral blood

Pharmacokinetics (PK) of FT839 in peripheral blood including but not limited to maximum concentration (Cmax) and area under the curve (AUC) will be estimated based on observed plasma concentration-time data.

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: Incidence of AEs and SAEs

Incidence and severity of AEs and SAEs

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: Cmax of FT839 in peripheral blood

PK of FT839 in peripheral blood including but not limited to Cmax will be estimated based on observed plasma concentration-time data

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years

Phase 2: AUC of FT839 in peripheral blood

PK of FT839 in peripheral blood including AUC will be estimated based on observed plasma concentration-time data

Time frame: From enrollment to the end of the post-treatment follow-up at 2 years