Safety and Clinical Activity of QEL-001 in A2-mismatch Liver Transplant Patients

This study is a multicenter, first-in-human, open-label, single-arm study of an autologous CAR T regulatory (CAR-Treg) in HLA-A2 mismatched liver transplant recipients. The aim is for the CAR-Tregs to be activated on recognition of HLA-A2 antigens present on the donated liver and subsequently induce and maintain immunological tolerance to the organ.

Inclusion criteria

• Written informed consent.
• Subject who are HLA A2/A28 negative who have received HLA A2-mismatch liver transplant 12 months to 5 years prior to study entry.
• Able and willing to use contraception.
• Be on stable maintenance of immunosuppression for at least 12 weeks prior to study entry.

Exclusion criteria

• Severe cardiac, respiratory disease or any other major organ dysfunction.
• Subjects with prior non-liver solid organ or hematopoietic stem cell transplant.
• Known hypersensitivity to study medication ingredients, protocol defined immunosuppressive medications, or a significant allergic reaction to any drug.
• Positive serology for human immunodeficiency virus (HIV), active or latent tuberculosis (TB) or other clinically active local or systemic infection.
• Use of investigational agents within 3 months of screening.
• Subjects with history of autoimmune disease requiring use of immunosuppression or biologics within 24 months prior to study entry.
• Subject with history of malignancy in the past 5 years.
• Medical or social condition that is not compatible with adequate study follow-up and any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study.
• Protocol defined laboratory value for the following parameters:

• Alanine aminotransferase (ALT) and either alkaline phosphatase (ALP) or gamma-glutamyl transferase (GGT),
• Kidney function e.g. eGFR,
• White blood cells,
• Hemoglobin,
• Platelets.