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

Официальное название

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)

Обзор

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.

Подробное описание

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

Вмешательства

• Биопрепарат AZD0120
  Single infusion of AZD0120 on visit DAY 1 after completing required lymphodepleting chemotherapy..
• Препарат Cyclophosphamide
  Lymphodepletion - specified dose prior to receiving AZD0120
• Препарат Fludarabine
  Lymphodepletion - specified dose prior to receiving AZD0120

Первичные конечные точки

• PHASE 1B: To evaluate the safety and tolerability of AZD0120 in participants with refractory systemic lupus erythematosus (SLE) [Срок оценки: 2 years]
• PHASE 1B: Recommended Phase 2 Dose (RP2D) of AZD0120 in for Phase 2 [Срок оценки: 2 years]
• PHASE 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]

Вторичные конечные точки (12)

• PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To evaluate the efficacy of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]
• PHASE 1B & 2: To characterize the cellular kinetics of AZD0120 in participants with refractory SLE [Срок оценки: 2 years]

Критерии участия

INCLUSION:

• Males or females aged 18 through 70 years inclusive at the time of consent.
• Written informed consent in accordance with federal, local, and institutional guidelines.
• Must be able and willing to adhere to the study visit schedule and other protocol requirements
• Adequate hepatic, renal, pulmonary, and cardiac function
• 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.
• Have used at least two standard immunosuppressants (including one biological agent).
• SLEDAI-2K score ≥6 at screening.
• Must include a significant SLE related organ involvement: arthritis, myositis, rash, alopecia, mucosal ulcers, pleurisy, pericarditis, vasculitis, or renal.
• 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:

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 15 центров

• Research Site — San Francisco
• Research Site — Stanford
• Research Site — Tampa
• Research Site — Atlanta
• Research Site — Chicago
• Research Site — Bethesda
• Research Site — Boston
• Research Site — St Louis
• … и ещё 7 центров

Австралия · 2 центра

• Research Site — Darlinghurst
• Research Site — Melbourne

Канада · 1 центр

• Research Site — Toronto

Идентификаторы

NCT: NCT06897930 · D8313C00001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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