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

Systemic Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06328777
• Other study ID #: CAB-201-003
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: June 2024
• Est. completion date: July 2029

Study information

• Verified date: June 2024
• Source: Cabaletta Bio
• Contact: Cabaletta Bio
• Phone: +1 267 759 3100
• Email: clinicaltrials[@]cabalettabio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: July 2029
• Est. primary completion date: July 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

Inclusion Criteria:

• Age =18 and =70

• 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

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.

Related Conditions & MeSH terms

• Scleroderma
• Scleroderma, Diffuse
• Scleroderma, Systemic
• Sclerosis
• Systemic Sclerosis

Intervention

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

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan

Sponsors (1)

Lead Sponsor	Collaborator
Cabaletta Bio

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate incidence of adverse events	Incidence and severity of AEs	Up to 28 days after CABA-201 infusion
Secondary	To evaluate adverse events and laboratory abnormalities	Incidence and severity of AEs, including changes in laboratory values and vital signs	Up to 156 weeks
Secondary	To characterize the pharmacodynamics (PD)	Levels of B cells in the blood	Up to 156 weeks
Secondary	To characterize the pharmacokinetics (PK)	Levels of CABA-201-positive T cells in the blood	Up to 156 weeks
Secondary	To evaluate efficacy	Proportion of subjects achieving revised CRISS criteria	Up to 156 weeks