Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria

Austin G Kulasekararaj; Anna-Elina Lehtinen; Cecily Forsyth; Shreyans Gandhi; Morag Griffin; Sixten Körper; Gabor Mikala; Petra Muus; Ulrik Overgaard; Christopher J Patriquin; Humphrey Pullon; Yu-Min Shen; Ruth Spearing; Jeff Szer; Guillemette De la Borderie; Petra W Duda; Ramin Farzaneh-Far; Sharan Ragunathan; Camil E Sayegh; Douangsone D Vadysirisack; Hubert Schrezenmeier

doi:10.3324/haematol.2022.281780

Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria

Haematologica. 2024 Mar 1;109(3):929-935. doi: 10.3324/haematol.2022.281780.

Authors

Austin G Kulasekararaj¹, Anna-Elina Lehtinen², Cecily Forsyth³, Shreyans Gandhi¹, Morag Griffin⁴, Sixten Körper⁵, Gabor Mikala⁶, Petra Muus⁷, Ulrik Overgaard⁸, Christopher J Patriquin⁹, Humphrey Pullon¹⁰, Yu-Min Shen¹¹, Ruth Spearing¹², Jeff Szer¹³, Guillemette De la Borderie¹³, Petra W Duda¹⁴, Ramin Farzaneh-Far¹⁴, Sharan Ragunathan¹⁴, Camil E Sayegh¹⁴, Douangsone D Vadysirisack¹⁴, Hubert Schrezenmeier¹⁵

Affiliations

¹ King's College Hospital-NHS Foundation Trust, NIHR/Wellcome King's Clinical Research Facility, London, UK and King's College London, London.
² Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki.
³ Gosford Hospital, Gosford, NSW.
⁴ St. James's University Hospital, Leeds.
⁵ University of Ulm, Institute of Transfusion Medicine, and Institute of Transfusion Medicine and Immunogenetics Ulm, Red Cross Blood Transfusion Service Baden-Württemberg-Hessen and University Hospital Ulm, Ulm.
⁶ Central Hospital of Southern Pest National Institute of Hematology and Infectious Diseases, Budapest.
⁷ King's College Hospital-NHS Foundation Trust, NIHR/Wellcome King's Clinical Research Facility, London, UK and King's College London, London, UK; St. James's University Hospital, Leeds.
⁸ University Hospital Copenhagen, Copenhagen, Denmark.
⁹ University Health Network, Toronto, ON.
¹⁰ Waikato Hospital, Hamilton.
¹¹ UT Southwestern Medical Center, Dallas, TX.
¹² Christchurch Hospital, Christchurch.
¹³ Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, Melbourne, VIC.
¹⁴ UCB Pharma, Cambridge, MA.
¹⁵ University of Ulm, Institute of Transfusion Medicine, and Institute of Transfusion Medicine and Immunogenetics Ulm, Red Cross Blood Transfusion Service Baden-Württemberg-Hessen and University Hospital Ulm, Ulm. h.schrezenmeier@blutspende.de.

No abstract available

Publication types

Clinical Trial, Phase II
Letter

MeSH terms

Complement C5* / therapeutic use
Hemoglobinuria, Paroxysmal* / drug therapy
Humans
Peptides, Cyclic* / therapeutic use

Substances

Complement C5
Peptides, Cyclic
zilucoplan

Grants and funding

Funding: Studies 201, 202, and 203 were funded by Ra Pharmaceuticals (Cambridge, MA, USA; now a part of UCB Pharma [Brussels, Belgium]). Medical writing and editorial assistance were provided by Jessica Deckman, PhD, CMPP, of The Lockwood Group (Stamford, CT, USA), and funded by UCB Pharma, in accordance with Good Publications Practice (GPP) 2022 guidelines (https://www.ismpp.org/gpp-2022).