Clinical trial NCT06897930: A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE
Official title: A Phase 1b/2 Study of AZD0120, a Chimeric Antigen Receptor T-cell (CAR T) Therapy Targeting CD19 and B-cell Maturation Antigen (BCMA) in Subjects With Refractory Systemic Lupus Erythematosus (SLE)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Lupus Erythematosus, Systemic
Keywords: AZD0120
SLE
Systemic Lupus Erythematosus
Lupus
Lupus Nephritis
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2025-03-27
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Intervention model description: Drug: AZD0120 Single infusion of AZD0120
Primary purpose: TREATMENT

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

Eligibility criteria: INCLUSION:

1. Males or females aged 18 through 70 years inclusive at the time of consent.
2. Written informed consent in accordance with federal, local, and institutional guidelines.
3. Must be able and willing to adhere to the study visit schedule and other protocol requirements
4. Adequate hepatic, renal, pulmonary, and cardiac function
5. Have a clinical diagnosis of SLE according to the EULAR/ American College of Rheumatology (ACR) 2019 criteria with a positive ANA ≥1:80 and a score ≥10.
6. Have used at least two standard immunosuppressants (including one biological agent).
7. SLEDAI-2K score ≥6 at screening.
8. Must include a significant SLE related organ involvement: arthritis, myositis, rash, alopecia, mucosal ulcers, pleurisy, pericarditis, vasculitis, or renal.
9. For lupus nephritis: Diagnosis of proliferative lupus nephritis based on a renal biopsy obtained within 6 months prior to signing the informed consent form or during the screening period Class III, IV or V LN according to the WHO 2003 ISN/RPS classification.

EXCLUSION:

1. Have received prior treatment with CAR T therapy directed at any target.
2. Have received any therapy that is targeted to CD19 and/or BCMA
3. Received allogenic stem cell transplant or autologous stem cell transplant.
4. An active malignancy that is progressing or requires active treatment.
5. Primary immunodeficiency
6. Active viral or bacterial infection

Primary outcomes:
measure: PHASE 1B: To evaluate the safety and tolerability of AZD0120 in participants with refractory systemic lupus erythematosus (SLE)
timeFrame: 2 years
measure: PHASE 1B: Recommended Phase 2 Dose (RP2D) of AZD0120 in for Phase 2
timeFrame: 2 years
measure: PHASE 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE
timeFrame: 2 years
Secondary outcomes:
measure: PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 1B & 2: To assess immunogenicity of AZD0120
timeFrame: 2 years
measure: PHASE 1B & 2: To determine whether replication-competent lentivirus is present in participants that receive AZD0120
timeFrame: 2 years
measure: PHASE 2: To further characterize the safety of AZD0120 in participants with refractory SLE
timeFrame: 2 years
measure: PHASE 2: To assess changes from baseline for reported health-related quality of life, overall health status
timeFrame: 2 years
measure: PHASE 2: To assess changes from baseline for reported health-related quality of life, overall health status
timeFrame: 2 years
Other outcomes:
Locations:
city: New York
country: United States
facility: Research Site
state: New York
city: Syracuse
country: United States
facility: Research Site
state: New York
city: Seattle
country: United States
facility: Research Site
state: Washington
Study officials:
References: