A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE

Phase 1

Recruiting

• Conditions: Lupus Erythematosus, Systemic
• Interventions: Biological: AZD0120; Drug: Cyclophosphamide; Drug: Fludarabine

• Registration Number: NCT06897930
• Lead Sponsor: AstraZeneca

Brief Summary

This is a Phase 1b/2, single-arm, open-label, multi-center, clinical study of AZD0120, a CD19/BCMA dual CAR T cell therapy, to evaluate the safety, tolerability, and efficacy in adult participants with refractory Systemic Lupus Erythematosus.

Detailed Description

The study will enrol adult participants with refractory Systemic Lupus Erythematosus (SLE) The study will be run in 2 parts;

First part is Phase 1b during which the study aims to assess safety and tolerability of AZD0120 in patients in refractory SLE cohorts

Second part is Phase 2, during which the study aims to assess safety, tolerability and efficacy of the selected dose, following Phase 1b completion, in patients with refractory SLE

Study Timeline

• Study First Submitted: 2025-03-12
• Study First Submitted QC: 2025-03-20
• Study First Posted: 2025-03-27
• Study Start: 2025-04-21
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-05
• Last Update Posted: 2025-05-06
• Primary Completion: 2029-05-01
• Study Completion: 2029-05-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
AZD0120	AZD0120	Single dose of AZD0120
AZD0120	Cyclophosphamide	Single dose of AZD0120
AZD0120	Fludarabine	Single dose of AZD0120

Primary Outcome Measures

Name	Time	Method
PHASE 1B: To evaluate the safety and tolerability of AZD0120 in participants with refractory systemic lupus erythematosus (SLE)	2 years	The incidence and severity of adverse events (AEs)
PHASE 1B: Recommended Phase 2 Dose (RP2D) of AZD0120 in for Phase 2	2 years	To determine the recommended phase 2 dose (RP2D) of AZD0120
PHASE 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE	2 years	Proportion of participants achieving SRI-4 response

Secondary Outcome Measures

Name	Time	Method
PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE	2 years	Time of maximum observed blood concentration (Tmax)
PHASE 1B & 2: To assess immunogenicity of AZD0120	2 years	Presence of AZD0120 antibodies
PHASE 1B & 2: To determine whether replication-competent lentivirus is present in participants that receive AZD0120	2 years	Presence of replication-competent lentivirus (RCL)
PHASE 2: To further characterize the safety of AZD0120 in participants with refractory SLE	2 years	Further characterization of the safety of AZD0120 by measuring the incidence and severity of Treatment Emergent Adverse Events (TEAEs)
PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE	2 years	Time from AZD0120 infusion and time from response to first disease flare
PHASE 2: To assess changes from baseline for reported health-related quality of life, overall health status	2 years	To assess changes from baseline for FUNCTIONAL ASSESSMENT OF CHRONIC ILLNESS THERAPY (FACIT)

Trial Locations

Locations (1)

Research Site; 🇺🇸 Seattle, Washington, United States