A phase I study of recombinant human C1 inhibitor in asymptomatic patients with hereditary angioedema

Journal of Allergy and Clinical Immunology

Volumn 116, Issue 4, 2005, Pages 876-883

  Van Doorn, Martijn B A ^a   Burggraaf, Jacobus ^a   Van Dam, Tijtje ^b   Eerenberg, Anke ^c   Levi, Marcel ^d   Hack, Cornelis E ^c   Schoemaker, Rik C ^a   Cohen, Adam F ^a   Nuijens, Jan ^b  

^a CENTRE FOR HUMAN DRUG RESEARCH   (Netherlands)

^b Pharming Group NV   (Netherlands)

^c SANQUIN RESEARCH   (Netherlands)

^d ACADEMIC MEDICAL CENTER   (Netherlands)

Author keywords

C1 inhibitor; C4; Complement; Hereditary angioedema; Human; Pharmacodynamics; Pharmacokinetics

Indexed keywords

COMPLEMENT COMPONENT C1S INHIBITOR; RECOMBINANT COMPLEMENT COMPONENT C 1 S INHIBITOR; RECOMBINANT PROTEIN; UNCLASSIFIED DRUG;

ANGIONEUROTIC EDEMA; ARTICLE; CLINICAL ARTICLE; CLINICAL TRIAL; COMPLEMENT ACTIVATION; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG EFFECT; DRUG HALF LIFE; DRUG INFUSION; DRUG ISOLATION; DRUG SAFETY; DRUG TOLERABILITY; HUMAN; MAXIMUM TOLERATED DOSE; MILK; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL; RABBIT; TRANSGENIC ANIMAL;

ANGIONEUROTIC EDEMA; ANIMALS; ANIMALS, GENETICALLY MODIFIED; COMPLEMENT C1 INACTIVATOR PROTEINS; COMPLEMENT C4; FEMALE; HUMANS; INFUSIONS, INTRAVENOUS; MALE; RABBITS; RECOMBINANT PROTEINS; SERPINS;

EID: 25844432828     PISSN: 00916749     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jaci.2005.05.019     Document Type: Article

References (28)

1. M. Cicardi, and A. Agostoni Hereditary angioedema N Engl J Med 334 1996 1666 1667
2. N.S. Landerman, M.E. Webster, E.L. Becker, and H.E. Ratcliffe Hereditary angioneurotic edema. II. Deficiency of inhibitor for serum globulin permeability factor and/or plasma kallikrein J Allergy 33 1962 330 341
3. B.L. Zuraw, and J.G. Curd Demonstration of modified inactive first component of complement (C1) inhibitor in the plasmas of C1 inhibitor-deficient patients J Clin Invest 78 1986 567 575
4. E.W. Nielsen, H.T. Johansen, K. Hogasen, W. Wuillemin, C.E. Hack, and T.E. Mollnes Activation of the complement, coagulation, fibrinolytic and kallikrein-kinin systems during attacks of hereditary angioedema Scand J Immunol 44 1996 185 192
5. M. Cugno, J. Nuijens, E. Hack, A. Eerenberg, D. Frangi, and A. Agostoni Plasma levels of C1- inhibitor complexes and cleaved C1- inhibitor in patients with hereditary angioneurotic edema J Clin Invest 85 1990 1215 1220
6. P.J. Spath, B. Wuthrich, and R. Butler Quantification of C1-inhibitor functional activities by immunodiffusion assay in plasma of patients with hereditary angioedema - evidence of a functionally critical level of C1-inhibitor concentration Complement 1 1984 147 159
7. J.A. Gelfand, R.J. Sherins, D.W. Alling, and M.M. Frank Treatment of hereditary angioedema with danazol. Reversal of clinical and biochemical abnormalities N Engl J Med 295 1976 1444 1448
8. J.J. Zurlo, and M.M. Frank The long-term safety of danazol in women with hereditary angioedema Fertil Steril 54 1990 64 72
9. A. Agostoni, and M. Cicardi Hereditary and acquired C1-inhibitor deficiency: biological and clinical characteristics in 235 patients Medicine (Baltimore) 71 1992 206 215
10. K. Bork, M. Pitton, P. Harten, and P. Koch Hepatocellular adenomas in patients taking danazol for hereditary angio-oedema Lancet 353 1999 1066 1067
11. D. Crampon, R. Barnoud, M. Durand, D. Ponard, C. Jacquot, and J.J. Sotto Danazol therapy: an unusual aetiology of hepatocellular carcinoma J Hepatol 29 1998 1035 1036
12. K. Bork, and G. Witzke Long-term prophylaxis with C1-inhibitor (C1 INH) concentrate in patients with recurrent angioedema caused by hereditary and acquired C1-inhibitor deficiency J Allergy Clin Immunol 83 1989 677 682
13. A. Agostoni, L. Bergamaschini, G. Martignoni, M. Cicardi, and B. Marasini Treatment of Acute Attacks of Hereditary Angioedema with C1-Inhibitor Concentrate Ann Allergy 44 1980 299 301
14. A. Agostoni, M. Cicardi, M. Cugno, and E. Storti Clinical problems in the C1-inhibitor deficient patient Behring Inst Mitt 93 1993 306 312
15. A.T. Waytes, F.S. Rosen, and M.M. Frank Treatment of hereditary angioedema with a vapor-heated C1 inhibitor concentrate N Engl J Med 334 1996 1630 1634
16. M. Kunschak, W. Engl, F. Maritsch, F.S. Rosen, G. Eder, and G. Zerlauth A randomized, controlled trial to study the efficacy and safety of C1 inhibitor concentrate in treating hereditary angioedema Transfusion 38 1998 540 549
17. D.E. Visentin, W.H. Yang, and J. Karsh C1-esterase inhibitor transfusions in patients with hereditary angioedema Ann Allergy Asthma Immunol 80 1998 457 461
18. A. Agostoni, E. Aygoren-Pursun, K.E. Binkley, A. Blanch, K. Bork, and L. Bouillet Hereditary and acquired angioedema: problems and progress: proceedings of the third C1 esterase inhibitor deficiency workshop and beyond J Allergy Clin Immunol 114 suppl 2004 S51 S131
19. K. Koles, P.H. Van Berkel, F.R. Pieper, J.H. Nuijens, M.L. Mannesse, and J.F. Vliegenthart N- and O-glycans of recombinant human C1 inhibitor expressed in the milk of transgenic rabbits Glycobiology 14 2004 51 64
20. G.J. Wolbink, J. Bollen, J.W. Baars, R.J. ten Berge, A.J. Swaak, and J. Paardekooper Application of a monoclonal antibody against a neoepitope on activated C4 in an ELISA for the quantification of complement activation via the classical pathway J Immunol Methods 163 1993 67 76
21. R.C. Schoemaker, and A.F. Cohen Estimating impossible curves using NONMEM Br J Clin Pharmacol 42 1996 283 290
22. J.E. Gadek, S.W. Hosea, J.A. Gelfand, M. Santaella, M. Wickerhauser, and D.C. Triantaphyllopoulos Replacement therapy in hereditary angioedema - successful treatment of acute episodes of angioedema with partly purified C1-inhibitor N Engl J Med 302 1980 542 546
23. L. Bergamaschini, M. Cicardi, A. Tucci, M. Gardinali, D. Frangi, and C. Valle C1 INH concentrate in the therapy of hereditary angioedema Allergy 38 1983 81 84
24. K. Bork, and S.E. Barnstedt Treatment of 193 episodes of laryngeal edema with C1 inhibitor concentrate in patients with hereditary angioedema Arch Intern Med 161 2001 714 718
25. J.O. Minta The role of sialic acid in the functional activity and the hepatic clearance of C1-INH J Immunol 126 1981 245 249
26. D.H. Perlmutter, G.I. Glover, M. Rivetna, C.S. Schasteen, and R.J. Fallon Identification of a serpin-enzyme complex receptor on human hepatoma cells and human monocytes Proc Natl Acad Sci U S A 87 1990 3753 3757
27. J.H. Nuijens, C.C. Huijbregts, A.J. Eerenberg-Belmer, J.J. Abbink, R.J. Strack Van Schijndel, and R.J. Felt-Bersma Quantification of plasma factor XIIa-Cl-inhibitor and kallikrein-Cl-inhibitor complexes in sepsis Blood 72 1988 1841 1848
28. C.E. Hack, J. Paardekooper, R.J. Smeenk, J. Abbink, A.J. Eerenberg, and J.H. Nuijens Disruption of the internal thioester bond in the third component of complement (C3) results in the exposure of neodeterminants also present on activation products of C3. An analysis with monoclonal antibodies J Immunol 141 1988 1602 1609