RESET-SSc: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T Cell Therapy, in Subjects With Systemic Sclerosis

Официальное название

A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Subjects With Systemic Sclerosis

Обзор

RESET-SSc: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T cell therapy, in Subjects with Systemic Sclerosis

Подробное описание

Systemic sclerosis (SSc), also known as scleroderma , is a rare autoimmune disorder characterized by autoantibody production and abnormal B cell function. Though the cause of SSc is not well understood, it is thought to involve B cells that cause the body to attack different tissues in one's own body, causing skin and organ fibrosis. This study is being conducted to evaluate the safety and efficacy of an investigational therapy, CABA-201, a CD19-CAR T cell therapy, that can be given to patients with SSc who have active disease. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.

Вмешательства

• Биопрепарат CABA-201
  Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide

Первичные конечные точки

• To evaluate incidence of adverse events [Срок оценки: Up to 28 days after CABA-201 infusion]

Вторичные конечные точки (4)

• To evaluate adverse events and laboratory abnormalities [Срок оценки: Up to 156 weeks]
• To characterize the pharmacodynamics (PD) [Срок оценки: Up to 156 weeks]
• To characterize the pharmacokinetics (PK) [Срок оценки: Up to 156 weeks]
• To evaluate efficacy [Срок оценки: Up to 156 weeks]

Критерии участия

Критерии включения

• Age ≥18 and ≤75
• A clinical diagnosis of SSc, based on the 2013 American College of Rheumatology and European League Against Rheumatism classification criteria.
• Early active disease
• Evidence of significant skin, pulmonary, renal, or cardiac involvement

Критерии исключения

• Contraindication to leukapheresis
• History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites
• Active infection requiring medical intervention at screening visit
• Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections.
• Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures
• Severe lung or cardiac impairment
• Previous CAR T cell therapy
• Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant

Other protocol-defined inclusion/exclusion criteria may apply.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 10 центров

• Yale University — New Haven
• Mayo Clinic Florida — Jacksonville
• Northwestern Memorial Hospital — Chicago
• The University of Chicago Medical Center — Chicago
• University of Kansas Medical Center — Kansas City
• University of Michigan — Ann Arbor
• Mayo Clinic Rochester — Rochester
• Columbia University Irving Medical Center — New York
• … и ещё 2 центра

Идентификаторы

NCT: NCT06328777 · CAB-201-003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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