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

Phase 1

Recruiting

• Conditions: Systemic Sclerosis; Scleroderma
• Interventions: Biological: CABA-201

• Registration Number: NCT06328777
• Lead Sponsor: Cabaletta Bio

Brief Summary

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

Detailed Description

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.

Study Timeline

• Study First Submitted: 2024-03-19
• Study First Submitted QC: 2024-03-19
• Study First Posted: 2024-03-25
• Study Start: 2024-07-02
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-08
• Last Update Posted: 2025-01-09
• Primary Completion: 2029-07
• Study Completion: 2029-07

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• 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

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Exclusion Criteria

• 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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CABA-201	CABA-201	Severe Skin Cohort: Infusion of CABA-201 with preconditioning in subjects with SSc with severe skin involvement Organ Cohort: Infusion of CABA-201 with preconditioning in subjects with SSc with organ involvement

Primary Outcome Measures

Name	Time	Method
To evaluate incidence of adverse events	Up to 28 days after CABA-201 infusion	Incidence and severity of AEs

Secondary Outcome Measures

Name	Time	Method
To characterize the pharmacokinetics (PK)	Up to 156 weeks	Levels of CABA-201-positive T cells in the blood
To evaluate efficacy	Up to 156 weeks	Proportion of subjects achieving revised CRISS criteria
To evaluate adverse events and laboratory abnormalities	Up to 156 weeks	Incidence and severity of AEs, including changes in laboratory values and vital signs
To characterize the pharmacodynamics (PD)	Up to 156 weeks	Levels of B cells in the blood

Trial Locations

Locations (5)

Northwestern Memorial Hospital; 🇺🇸 Chicago, Illinois, United States

The University of Chicago Medical Center; 🇺🇸 Chicago, Illinois, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Columbia University Irving Medical Center; 🇺🇸 New York, New York, United States

Duke University; 🇺🇸 Durham, North Carolina, United States