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

NCT03995901 | Terminated Phase 3 DMC FDA Drug

• 1 other identifier: FCR001A2301
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 18

Brief Summary

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

Conditions

Transplanted Organ Rejection

Keywords

Kidney Transplant; Stem cell therapy; Anti-rejection medications; Living donor kidney transplant; Immune tolerance; Immunosuppression; Immunosuppressive medication; Organ transplant rejection

Outcome Measures

Primary Outcomes (1)

• Proportion of FCR001 recipients who are free from immunosuppression (IS), without biopsy proven acute rejection (BPAR) at 24 months post-transplant

  Free from IS is defined as not taking any immunosuppression medications and not having to take immunosuppression medications since their withdrawal. Biopsy proven acute rejection is defined as Grade ≥1A according to the Banff 2017 Classification of Antibody-Medicated Rejection and T Cell-Mediated Rejection in Renal Allografts (Haas et al 2018).

  24 months post-transplant

Secondary Outcomes (33)

• Change in renal function by Modification of Diet in Renal Disease (MDRD4) from post-transplant baseline (Month 1) to Month 24 in FCR001 recipients

  24 months post-transplant

• Proportion of FCR001 recipients free from IS, without BPAR, at Month 36 and 60

  Month 36 and 60 post transplant

• Allograft function (eGFR by MDRD4) and change in eGFR from Month 1 to Month 24, 36, and Month 60, by treatment

  Month 1 (post-transplant) to Month 24, 36, and Month 60

• Slope and difference in slope of estimated glomerular filtration rate (eGFR) by Modification of Diet in Renal Disease (MDRD4) over time to Month 24, 36, and 60, by treatment

  Month 24, 36, and 60

• Allograft function (eGFR) and change in renal allograft function from Month 1 to Months 24, 36 and 60 by treatment group, using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula

  Month 1 (post transplant) to Month 24, 36, and Month 60

Study Arms (2)

FCR001

EXPERIMENTAL

FCR001 is a cryopreserved allogeneic stem cell therapy derived from mobilized peripheral blood of the kidney donor that is delivered as a single dose with a non- myeloablative conditioning regimen. FCR001 contains the donor's CD34+ cells, facilitating cells, and αβ T cells.

Biological: FCR001

Control

NO INTERVENTION

Standard induction therapy followed by a maintenance regimen of tacrolimus, mycophenolate, and +/- corticosteroids after kidney transplant. Control donors are not followed beyond randomization.

Interventions

FCR001 BIOLOGICAL

FCR001 is a cryopreserved allogeneic stem cell therapy derived from mobilized peripheral blood of the kidney donor that is delivered as a single dose with a non- myeloablative conditioning regimen. FCR001 contains the donor's CD34+ cells, facilitating cells, and αβ T cells.

Also known as: Cell based therapy, Allogeneic stem cell transplant

FCR001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Talaris Therapeutics Inc.: lead

Study Sites (18)

Mayo Clinic

Phoenix, Arizona, 85054, United States

Location

Scripps Clinic

La Jolla, California, 92037, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

The University of Michigan Hospitals & Health System

Ann Arbor, Michigan, 48109, United States

Location

University of Minnesota Medical Center

Minneapolis, Minnesota, 55455, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

New York-Presbyterian/Weill Cornell

New York, New York, 10065, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

MeSH Terms

Interventions

Cell- and Tissue-Based Therapy

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2019
• First Posted: June 24, 2019
• Study Start: October 25, 2019
• Primary Completion: February 16, 2023
• Study Completion: February 16, 2023
• Last Updated: March 3, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (18)