A Safety and Efficacy Study of FCR001 vs Standard of Care in de Novo Living Donor Kidney Transplantation

A Randomized, Controlled, Multi-center, Safety and Efficacy Study of FCR001 Cell-based Therapy Relative to a Tacrolimus and Mycophenolate-based Regimen in de Novo Living Donor Renal Transplant Recipients, and Safety in FCR001 Donors

A randomized controlled study to evaluate the safety, efficacy, and overall benefit of FCR001 cell therapy in de novo living donor renal transplantation.

The purpose of this randomized (2:1) controlled study is to evaluate the safety, efficacy and overall benefit of FCR001 cell therapy in first or second de novo living donor renal transplantation relative to a standard-of-care control immunosuppression regimen of antibody induction, tacrolimus, mycophenolate, and corticosteroids.

Main Inclusion Criteria:

• Recipient age ≥18 years.
• Donor age ≥18 and ≤60 years at time of signing informed consent.
• Recipients of a first or second living donor kidney transplant
• Donor willing to undergo mobilization, apheresis and 12-month safety follow-up and meet all local standard eligibility criteria to donate stem cells for allogeneic transplantation.
• Recipient meets all local standard eligibility criteria for allogeneic stem cell transplant.
• Donors must be deemed eligible as per the requirements of 21CFR1271.

Main Recipient and Donor Exclusion Criteria:

• Recipient and donor who are identical twins.
• Recipient or donor with history of malignancy or premalignant syndrome (e.g., myelodysplastic syndrome, monoclonal gammopathy of renal significance [MGRS], monoclonal gammopathy of unknown significance [MGUS]) of any organ system (other than localized basal cell carcinoma of the skin or in-situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
• Recipient or donor with known bone marrow aplasia.

Main Recipient-only Exclusion Criteria:

• Multi-organ or stem cell transplant recipient.
• Calculated panel reactive antibodies >80%.
• Recipient is blood type ABO incompatible with donor.
• Presence of donor-specific antibodies (DSA) (positive result) at any time pre-transplant.
• Recipient who is human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) positive.
• Recipient with any baseline condition requiring or anticipated will require chronic or intermittent use of systemic steroids or other IS (eg, autoimmune disease, asthma) throughout the course of the study.
• Recipient with a BMI < 18 or > 35 kg/m2.
• Recipient requiring systemic anticoagulation, (eg, for hyper-coagulation disorders, deep vein thrombosis, atrial fibrillation) that cannot be temporarily interrupted which would preclude renal biopsy.

Main Donor-only Exclusion Criteria:

• Biologically unrelated (i.e., no genetic relationship) female donor transplant to male recipient.