Review
Copyright ©The Author(s) 2015.
World J Nephrol. May 6, 2015; 4(2): 169-184
Published online May 6, 2015. doi: 10.5527/wjn.v4.i2.169
Table 1 Representative abnormalities in complement leading to renal disease
Components/related moleculesDiseases
Complement C3C3 glomerulopathy (DDD), aHUS
Factor HC3 glomerulopathy (DDD/C3GN), aHUS
Factor IC3 glomerulopathy (C3GN), aHUS
MCPaHUS
Factor BaHUS
CFHR5Familial C3 glomerulopathy (CFHR5 nepropathy)
CFHR3-1Familial C3 glomerulopathy
CFHR1/3IgA nephropathy, aHUS
Factor B autoantibodyC3 glomerulopathy (DDD)
Factor H autoantibodyC3 glomerulopathy (DDD/C3GN)
Bb (activated factor B)HUS, ANCA-associated vasculitis
C3NefC3 glomerulopathy (DDD, C3GN)
Soluble C5b-9HUS, TTP, ANCA-associated vasculitis
C3aANCA-associated vasculitis, TTP
C5aANCA-associated vasculitis
C1q/C1qRC1q nephropathy
ProperdinTI injury due to massive proteinuria
C5ANCA-associated vasculitis
Factor BANCA-associated vasculitis
CRaRTI inflammation, IRI
C5aRIRI
Factor HIRI
C5b-9IRI
CD59IRI
Table 2 Some of the molecules aimed to target complement, phase of the trial and renal diseases related
Compound nameComplement targetCompound classPhase/indication
C1 inhibitor (Berinert)C1r, C1s, MASP1, MASP2RegulatorP 1/2 transplant
Cp40, AMY 101PC transplant , aHUS, DDD
sCR1, CDX-1135C3 conv, C4b, C3bRegulatorP 1 DDD
Mirococept, APT070C3 conv, C4b, C3bRegulatorP 1/2 transplant
EculizumabC5AbP 4 aHUS, P 2/3 STEC-HUS, P 2 ANCA vasculitis, P 1 transplant
MubodinaC5AbPC aHUS, DDD
ErgidinaC5AbPC transplant
CCX168C5aRSmall moleculeP 2 ANCA vasculitis
ADC-1004C5aRProteinPC transplant
Table 3 Trials ongoing with eculizumab in renal diseases and in renal transplantation
RankIdentifierStatusStudy name
1NCT01221181ActiveEculizumab therapy for dense deposit disease and C3 nephropathy
2NCT02093533RecruitingEculizumab in primary MPGN
3NCT01275287ActiveTargeting complement activation in ANCA-vasculitis
4NCT00844545CompletedOpen label controlled trial of eculizumab in adult patients with plasma therapy-resistant aHUS
5NCT00844844CompletedOpen label controlled trial of eculizumab in adolescent patients with plasma therapy-resistant aHUS
6NCT00844428UnknownOpen label controlled trial of eculizumab in adolescent patients with plasma therapy-sensitive aHUS
7NCT00838513UnknownOpen label controlled trial of eculizumab in adult patients with plasma therapy-sensitive aHUS
8NCT01194973UnknownAn open-label, multi center clinical trial of eculizumab in adult patients with aHUS
9NCT01193348UnknownAn open label, Multi center clinical trial of eculizumab in pediatric patients with aHUS
10NCT01755429UnknownThe safety and efficacy of eculizumab in Japanese patients with aHUS
11NCT01522170EnrollingaHUS observational long term follow up
12NCT01522183RecruitingaHUS registry
13NCT01770951CompletedA retrospective, observational, non-interventional trial to assess eculizumab treatment effect in patients with aHUS
14NCT02205541Not yet recruitingEculizumab in shiga-toxin related hemolytic and uremic syndrome pediatric patients
15NCT01410916CompletedSafety and efficacy study of eculizumab in shiga-toxin producing Escherichia coli (STEC-HUS)
16NCT01406288CompletedCompleted outbreak of HUS linked to Escherichia coli of serotype 0104:H4
17NCT01756508RecruitingEculizumab for prevention and treatment of kidney graft reperfusion injury
18NCT01919346RecruitingEculizumab for prevention of DGF in kidney transplantation
19NCT01403389ActiveA study of the activity of eculizumab for prevention of DGF in deceased donor transplant
20NCT02142182RecruitingA trial for prevention of DGF after kidney transplantation
21NCT01567085ActiveSafety and efficacy of eculizumab in the prevention of AMR in sensitized recipients of a kidney transplant from a deceased donor
22NCT02113891Not yet recruitingEculizumab therapy for subclinical antibody-mediated rejection in kidney transplantation
23NCT01095887ActiveEculizumab added to conventional treatment in the prevention of antibody-mediated rejection in blood group incompatible living donor kidney transplantation
24NCT01106027ActiveDosing regimen of eculizumab added to conventional treatment in positive crossmatch deceased kidney yransplant
25NCT01895127RecruitingEfficacy and safety of eculizumab for treatment of antibody-mediated rejection following renal transplantation
26NCT00670774ActiveDosing regimen of eculizumab added to conventional treatment in positive crossmatch living kidney transplant
27NCT01399593ActiveSafety and efficacy of eculizumab to prevent AMR in living donor kidney transplant recipients receiving desensitization
28NCT01327573ActiveEculizumab therapy for chronic complement-mediated injury in kidney transplantation
29NCT01029587RecruitingEculizumab to enable renal transplantation in patients with history of catastrophic antiphospholipid antibody syndrome