A Study to Test CRT-402 in Refractory Autoimmune Disease

A Phase 1/2 Dose Escalation and Expansion Study of CRT-402, an In Vivo CD19 Targeted CAR-T Therapy, in Refractory Autoimmune Disease

Patients with refractory autoimmune diseases often have limited treatment options and ongoing disease activity despite standard therapies. CRT-402 is an in vivo Cluster of differentiation 19 (CD19)-targeted CAR-T cell therapy designed to deplete CD19-positive B cells and promote immune system reset. This study evaluates the safety, tolerability, preliminary efficacy, pharmacodynamics (PD), and pharmacokinetics (PK) of CRT-402 in participants with active refractory systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and idiopathic inflammatory myopathies (IIM).

This is a multicenter, open-label, Phase 1/2, first-in-human, dose escalation and expansion study designed to assess the safety and tolerability, as well as define the recommended Phase 2 dose (RP2D) of CRT-402 in participants with refractory autoimmune disease.

Inclusion Criteria:

• Age 18 years or older.
• Diagnosis of active, refractory systemic lupus erythematosus, systemic sclerosis, or idiopathic inflammatory myopathy meeting established classification criteria, with inadequate response or intolerance to standard therapy.
• Adequate renal, hepatic, cardiac, and pulmonary function per protocol-defined criteria.
• Participants of reproductive potential must agree to use protocol-specified contraception during the study and for a defined period after dosing.
• Females of childbearing potential must have a negative pregnancy test before treatment.
• Must be willing and able to attend study visits and follow all study requirements.

Exclusion Criteria:

• Clinical suitability for a less burdensome and/or approved therapeutic approach, as judged by the Investigator,
• Any medical condition or laboratory abnormality that, in the Investigator's judgment, would place the participant at unacceptable risk or confound interpretation of study data,
• Prior Cluster of differentiation 19 (CD19)-directed, cell, or gene therapy,
• History of bone marrow/ hematopoietic stem cell or solid organ transplantation,
• Active or inadequately treated infection, including Human immunodeficiency (HIV), hepatitis B or C, or tuberculosis,
• Pregnancy or lactation.