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CINRYZE as a Donor Pre-treatment Strategy in Kidney Recipients of KDPI>60%

Phase 1
Conditions
Kidney Failure
Interventions
Drug: Placebo saline solution
Registration Number
NCT02435732
Lead Sponsor
University of Wisconsin, Madison
Brief Summary

Limiting brain death-induced organ injury through a systemic anti- inflammatory medical management should allow for improvement in the quality of transplanted organs, and as a result, clinical improvement in post-transplant outcomes represented by a decrease in the incidence of delayed graft function (DGF) after transplantation.

The specific aim is to evaluate the effect of C1INH (CINRYZE) as a donor pre-treatment strategy to decrease systemic inflammation and decrease the incidence of DGF in Expanded Criteria Donors (ECD), currently identified as donors with Kidney Donor Profile Index (KDPI) greater than or equal to 60%.

Detailed Description

This is a randomized, single-center double-blinded study.

Donor Pre-treatment Strategy:

The main objective is to identify the lowest dose that will allow an at least 80% decrease in the activity of classic pathway and Mannose-Binding Lectin (MBL) pathway of complement. The main objectives parallel observations in non-human primates in which animals receiving kidneys from donors in which activity of both classic and MBL pathways of complement were reduced by at least 50% with the use of C1 inhibitors displayed very mild or no delayed graft function after transplantation.

This trial has specifically been designed to evaluate the beneficial effect of C1INH in kidneys from deceased donors which have a high rate of delayed graft function. The selection of potential donors to be part of this study will be limited to the population of donors with a KDPI over 60%.

A total of 36 brain dead donors and 72 kidney recipients will be included in the study.

Most of the donors with a Kidney Donor Profile Index (KDPI) \>60%, due to the fact that they are considered "extreme" donors, are not likely to be able to donate organs other than kidneys. They would certainly not be pancreas donors, and it is unlikely they would be lung donors. There is a small possibility that donors with a KDPI \>60% could be potential liver donors. In the event that a donor liver is available for transplant, we will obtain written consent from the liver recipient, as we will from the kidney recipient(s) before the donor is dosed with the CINRYZE/placebo.

For this study:

All donors will come from within our service area. All kidney and livers will be allocated to be transplanted at the UW.

Stage 1: Collection of initial safety data prior to expanding the study to a broad cohort of patients.

3 non-randomized donors: Step 1: Two kidney only donors treated with 200 units/kg C1INH and heparin at 20 units/kg/h IV maintenance until organ recovery Step 2: Two donors of both liver and kidneys, treated with 200 units/kg C1INH and heparin at 20 units/kg/h IV maintenance until organ recovery The 8 kidneys and 2 livers in Stage 1 will be allocated to be transplanted only at UW.

Stage 2: PK Study, Safety and Outcome Data

36 donors will be randomized into 3 groups:

Group 1: Control group: standard donor management + vehicle treatment (n=12) Group 2: Standard donor management + C1INH at a dose of 200 U/Kg IV single dose (n=12) Group 3: Standard donor management + C1INH at a dose of 200 units/kg IV single dose and heparin at 20 units/kg/h IV maintenance until organ recovery (n=12)

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
72
Inclusion Criteria

Not provided

Exclusion Criteria
  • Use of an investigational drug in the 30 days before surgery.
  • Participation in any other research study (drug or non-drug) without prior approval from the Medical Monitor.
  • Known hypersensitivity to human monoclonal antibodies or any of the study drug excipients.
  • Previous hypersensitivity to basiliximab, Campath-1H or antithymocyte globulin (ATG)
  • History of or known HIV, HBV (surface antigen), or HCV positivity
  • History of malignancy within the last five years, except excised squamous or basal cell carcinoma of the skin, or cervical intraepithelial neoplasia.
  • Scheduled to undergo multi-organ transplantation.
  • Presence of clinically significant infections requiring continued therapy.
  • Positive screening for active tuberculosis.
  • Existence of any surgical or medical condition, other than the current transplantation which, in the opinion of the investigator, might significantly alter the distribution, metabolism or excretion of study medication.
  • History or presence of a medical condition or disease that in the investigator's assessment would place the patient at an unacceptable risk for study participation.
  • Lactating or pregnant woman.
  • Patient institutionalized by administrative or court order.
  • HLA or ABO incompatible kidney defined as a positive cytotoxic crossmatch, positive mean fluorescent intensity beyond the acceptable parameters by the institution, or flow crossmatch-based assay that is positive (for kidney recipients only)

Exclusion Criteria for Brain Dead Donor:

  • Donor who is known to have received an investigational drug for I-R injury or graft rejection (immunosuppressant) in the 48H prior to organ recovery
  • Participation in any other research study (drug or non-drug) without prior approval from the PI
  • Donor institutionalized by administrative or court order
  • Donors whose organs are allocated for transplantation to other transplant programs outside UW
  • Donors for which any of the intended organ recipients has not provided consent for the study
  • Donors that are donating other organs outside the scope of the study (i.e. heart, lungs, intestine) will be excluded.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
200 units/kg IV CINRYZE with Heparin 20 U/kg/h IVCINRYZECINRYZE 200 units/kg IV single dose with Heparin at 20 units/kg/h IV maintenance until organ recovery
200 units/kg IV CINRYZE with Heparin 20 U/kg/h IVPlacebo saline solutionCINRYZE 200 units/kg IV single dose with Heparin at 20 units/kg/h IV maintenance until organ recovery
Control groupPlacebo saline solutionStandard donor management + vehicle treatment (n=9)- placebo saline solution
CINRYZE 200 U/Kg IVPlacebo saline solutionIntervention is CINRYZE 200 U/Kg IV single dose
CINRYZE 200 U/Kg IVCINRYZEIntervention is CINRYZE 200 U/Kg IV single dose
200 units/kg IV CINRYZE with Heparin 20 U/kg/h IVHeparinCINRYZE 200 units/kg IV single dose with Heparin at 20 units/kg/h IV maintenance until organ recovery
Primary Outcome Measures
NameTimeMethod
Lowest dose that will allow at least an 80% decrease in the activity of classic pathway and MBL pathway of complement in brain death donors with KDPI over 60%, with the purpose of reducing the incidence of delayed graft function.over a 12 month period

Assessment of graft function

Secondary Outcome Measures
NameTimeMethod
Rate of of DGF in kidney transplantation from ECD brain death donorsWithin 6 hours of brain death

DGF rate

Donor Pharmacokinetics: plasma concentrations and Area under the Curve (AUC) for CINRYZEAt various timepoints within first 24 hours

pharmacokinetics

Level of suppression of the classical and MBL pathwaysWithin 6 hours of brain death

classical and MBL pathways

Number of Participants with Adverse Events as a Measure of Safety and TolerabilityDay of Transplant: first 24 hours, days 2-7, weeks 2,3,8, month 3, 6, 12

Safety and tolerability

Levels of complement activation after brain death in donors treated with C1INHWithin 6 hours of brain death

Complement activation

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