A Study of AZD0120 in Autoimmune Diseases (AURORA)

A Phase 1b, Open-label, Multi-cohort Study of AZD0120, an Autologous CD19/BCMA Targeting Chimeric Antigen Receptor T-cell, in Adults With Autoimmune Diseases

This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM), or difficult-to-treat rheumatoid arthritis (D2T RA).

Study Overview

• Status: Recruiting
• Conditions: Rheumatoid Arthritis; Systemic Sclerosis; Idiopathic Inflammatory Myopathies
• Intervention / Treatment: Biological: AZD0120

Detailed Description

This is a Phase 1b, open-label, multi-center, multi-cohort clinical study of AZD0120, a CD19/BCMA dual CAR T-cell therapy, to evaluate the safety in adult participants with systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM), or difficult-to-treat rheumatoid arthritis (D2T RA) for determination of the recommended phase 2 dose for each disease cohort. Approximately 9-12 participants will be evaluated per disease cohort.

• Study Type: Interventional
• Enrollment (Estimated): 27
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: AstraZeneca Clinical Study Information Center; Phone Number: 1-877-240-9479; Email: information.center@astrazeneca.com

Study Locations

• Australia
  • Darlinghurst, Australia, 2010
    • Recruiting
    • Research Site
  • Waratah, Australia, 2298
    • Not yet recruiting
    • Research Site
• Germany
  • Hamburg, Germany, 20246
    • Not yet recruiting
    • Research Site
  • Mainz, Germany, 55131
    • Not yet recruiting
    • Research Site
  • Würzburg, Germany, 97080
    • Not yet recruiting
    • Research Site
• Spain
  • Barcelona, Spain, 8035
    • Not yet recruiting
    • Research Site
  • Madrid, Spain, 28046
    • Not yet recruiting
    • Research Site
  • Madrid, Spain, 28041
    • Not yet recruiting
    • Research Site
• United Kingdom
  • Edinburgh, United Kingdom, EH4 2XU
    • Not yet recruiting
    • Research Site
  • London, United Kingdom, SE5 9RS
    • Not yet recruiting
    • Research Site
• United States
  • Arizona
    • Tucson, Arizona, United States, 85719
      • Not yet recruiting
      • Research Site
  • California
    • Stanford, California, United States, 94305-5847
      • Not yet recruiting
      • Research Site
  • Illinois
    • Chicago, Illinois, United States, 60637
      • Not yet recruiting
      • Research Site
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Not yet recruiting
      • Research Site
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Not yet recruiting
      • Research Site
  • New York
    • New York, New York, United States, 10032
      • Recruiting
      • Research Site
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Not yet recruiting
      • Research Site
  • Washington
    • Seattle, Washington, United States, 98104
      • Not yet recruiting
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Capable of giving signed informed consent.
• Adequate physiological function and reserve at screening.
• Able to comply with recommended medication washout period.
• Participants who are suitable for the study as determined by medical evaluation and at the discretion of the investigator.
• Willingness to remain on/start appropriate, highly effective methods of birth control or other acceptable criteria.

Exclusion Criteria:

• BMI at screening < 18 or > 35kg/m2.
• Any prior CAR T exposure.
• Unable or unwilling to remain within proximity (~2 hours travel time) of the administering investigational site for the first 28 days post study drug administration.
• Received a bone marrow or solid organ transplant at any time or on an active transplant waiting list.
• Received any investigational drug within ≥ 5 half-lives or 4 weeks, whichever is longer, prior to screening.
• Has certain heart conditions that could make it unsafe or unsuitable to take part in the study.
• Requirement for supplemental oxygen at rest (except at night for sleep apnea) or mechanical ventilation.
• Uncontrolled hypertension (> 160/100 mmHg) or symptomatic hypertension.
• Any central nervous system disease that may impact participants safety in the investigator's opinion.
• Other concurrent autoimmune or autoinflammatory disease. Certain autoimmune/autoinflammatory diseases may be included after discussion with the medical monitor.
• Evidence of clinically significant bleeding or active bleeding conditions within 90 days before screening
• History of malignancy or ongoing treatment for prior malignancy. Certain malignancies may be excepted.
• Known genetic inborn error of immunity and/or primary immunodeficiency.
• Active viral, bacterial, or fungal infection, or any ongoing infection that requires systemic antimicrobial therapy in the 4 weeks prior to screening.
• Seropositive for HIV.
• Active viral hepatitis are excluded.
• Active syphilis, positive for Treponema pallidum antibody.
• Vaccinated with live, attenuated vaccine within 4 weeks prior to apheresis or lymphodepletion.
• Not up-to-date on vaccinations per local/national health authority or institutional guidelines for immune-compromised individuals.
• Known life threatening allergies, hypersensitivity, or intolerance to AZD0120 or its excipients, including dimethyl sulfoxide.
• Contraindications or hypersensitivity to fludarabine and cyclophosphamide.
• Major surgery, or has surgery planned during the study.
• Pregnant, breastfeeding, or planning to become pregnant while enrolled in this study or within 1 year after receiving study treatment (whichever is later).
• Plans to father a child while enrolled in this study or within 1 year after receiving study treatment (whichever is later).
• Any issue that would impair the ability of the participant to receive or tolerate the planned treatment at the investigational site, to provide informed consent or any condition in the opinion of the investigator, participation would not be in the best interest of the participant.

Other protocol-defined eligibility criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AZD0120 Regimen 1; Participants will receive an infusion of AZD0120 Regimen 1.	Biological: AZD0120; CD19/BCMA Autologous CAR T-cell therapy product; Other Names: GC012F
Experimental: AZD0120 Regimen 2; Participants will receive an infusion of AZD0120 Regimen 2.	Biological: AZD0120; CD19/BCMA Autologous CAR T-cell therapy product; Other Names: GC012F

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants and severity of dose limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs)	Incidence and severity of DLTs and TEAEs to evaluate the safety of AZD0120 and to confirm the recommended Phase 2 dose (RP2D) in each indication SSc, IIM, or RA	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cellular Kinetics - Cmax	The maximum concentration of CAR T cells achieved in the body.	1 year
Cellular Kinetics - Tmax	The time point at which the Cmax is reached	1 year
Cellular Kinetics - AUC	The area under the concentration-time curve, which represents the total drug exposure over a specific period.	1 year
Cellular Kinetics - t½λz	The terminal elimination half-life.	1 year
Cellular Kinetics - Clast	The last observed quantifiable concentration of CAR T-cells.	1 year
Cellular Kinetics - Tlast	The time to last quantifiable concentration.	1 year
Cellular Kinetics - AUClast	The area under the concentration-time curve to the last measurable concentration.	1 year
Anti-drug antibodies (ADA) developed against AZD0120 from baseline	Humoral immunogenicity assessment of AZD0120 in participants with SSc, IIM, or RA.	1 year
Proportion of participants with detectable replication competent lentivirus (RCL) at pre-specified post infusion timepoints.	Incidence of vector-derived RCL in participant receiving AZD0120.	1 year
Change from baseline in the Disease Activity Score (DAS) 28-C-reactive protein (CRP). The DAS28-CRP is a measure from 0-10 with higher scores indicating greater disease activity.	Disease activity measures in RA participants.	1 year
Change from baseline in modified Rodnan Skin Score (mRSS). The mRSS is a measure of skin thickness with a range of 0-51 with higher scores indicating more severe disease.	Disease activity measures in SSc participants.	1 year
Change from baseline in the total improvement score (TIS). The TIS ranges from 0-100 with higher scores indicating greater improvement.	Disease activity measures in IIM participants.	1 year

Collaborators and Investigators

• Sponsor: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Actual): January 9, 2026
• Primary Completion (Estimated): February 22, 2028
• Study Completion (Estimated): February 22, 2028

Study Registration Dates

• First Submitted: October 16, 2025
• First Submitted That Met QC Criteria: December 19, 2025
• First Posted (Actual): December 22, 2025

Study Record Updates

• Last Update Posted (Actual): June 2, 2026
• Last Update Submitted That Met QC Criteria: June 1, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: CAR-T; Rheumatoid Arthritis; CD19; Systemic Sclerosis; BCMA; Idiopathic Inflammatory Myopathies
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Arthritis; Joint Diseases; Rheumatic Diseases; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Scleroderma, Systemic; Arthritis, Rheumatoid; Myositis
• Other Study ID Numbers: D8318C00001; 29707 (Other Identifier: IND)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

• IPD Sharing Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• IPD Sharing Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No