Phase III study to evaluate efficacy and safety of C1-INH
Research type
Research Study
Full title
A double-blind, randomised-withdrawal, placebo-controlled study to evaluate the efficacy and safety of human plasma-derived C1-esterase inhibitor as add-on to standard of care for the treatment of refractory antibody mediated rejection in adult renal transplant recipients.
IRAS ID
227736
Contact name
Nizam Mamode
Contact email
Sponsor organisation
CSL Behring LLC
Eudract number
2017-000348-17
Duration of Study in the UK
4 years, 1 months, 1 days
Research summary
Approximately 90 subjects will be enrolled across 25 hospitals who have been diagnosed with refractory antibody mediated rejection following a kidney transplant.
Antibody mediated rejection (AMR): After a kidney has been transplanted some patients experience episodes where the body tries to reject the new kidney as it recognises that it is different. In some cases this happens through the creation of antibodies that act against the kidney. There are recognised treatments to try and suppress this reaction but they do not work in all cases, in which case the AMR is termed refractory.
This study will evaluate the efficacy and safety of C1-INH in the treatment of refractory AMR following a kidney transplant.
The study consists of 2 treatment periods, and a post-treatment follow-up period:
• Treatment Period 1: Eligible subjects will receive a human plasma derived C1-esterase Inhibitor (C1-INH) at a dose of 60 IU/kg for 13 weeks. Subjects who respond to treatment with C1-INH by Week 12 as assessed by pre-defined responder criteria will be randomised into Treatment Period 2 of the study; subjects who do not respond will discontinue participation at Week 13.
• Treatment Period 2: Subjects who are responders will be randomised to receive either C1-INH or placebo (dummy drug) for 25 weeks. All subjects will receive standard of care throughout Treatment Periods 1 and 2 (IVIg 2 grams/kg once every 4 weeks; plasmapheresis as needed).
• At the end of Treatment Period 2 (Week 38), subjects will enter a 3.5-year post-treatment follow-up period, where they will receive treatment consistent with their local standard of care and they will be monitored for approximately 3.5 years.
• Subjects who experience a biopsy-proven AMR recurrence during the post-treatment follow-up will be permitted to undergo blinded retreatment according to their assigned treatment in Treatment Period 2, in combination with standard of care.REC name
London - Surrey Borders Research Ethics Committee
REC reference
17/LO/1022
Date of REC Opinion
19 Jan 2018
REC opinion
Further Information Favourable Opinion