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

AstraZeneca

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Lupus Erythematosus, Systemic

Treatments: Drug: Cyclophosphamide; Drug: Fludarabine; Biological: AZD0120

Study type: Interventional

Funder types: Industry

Identifiers

NCT06897930

D8313C00001

Details and patient eligibility

About

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.

Full 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

Enrollment: 150 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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150 participants in 1 patient group

AZD0120

Experimental group

Description:

Single dose of AZD0120

Treatment:

Biological: AZD0120

Drug: Fludarabine

Drug: Cyclophosphamide