Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia
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ROBAK, Tadeusz, KAZMIERCZAK, Maciej, JARQUE, Isidro, MUSTEAŢĂ, Vasile, TRELINSKI, Jacek, COOPER, Nichola, KIESSLING, Peter C., MASSOW, Ute, WOLTERING, Franz, SNIPES, Rose G., KE, Juan, LANGDON, Grant, BUSSEL, James Bruce, JOLLES, Stephen. Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia. In: Blood Advances, 2020, nr. 4(17), pp. 4136-4146. ISSN 2473-9537. DOI: https://doi.org/10.1182/bloodadvances.2020002003
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Blood Advances
Numărul 4(17) / 2020 / ISSN 2473-9537

Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia

DOI: https://doi.org/10.1182/bloodadvances.2020002003

Pag. 4136-4146

Robak Tadeusz12, Kazmierczak Maciej3, Jarque Isidro45, Musteaţă Vasile6, Trelinski Jacek12, Cooper Nichola7, Kiessling Peter C.8, Massow Ute8, Woltering Franz8, Snipes Rose G.9, Ke Juan10, Langdon Grant11, Bussel James Bruce12, Jolles Stephen13
 
1 Medical University of Lodz,
2 Copernicus Memorial Hospital, Lodz,
3 Poznan University of Medical Sciences,
4 La Fe University and Polytechnic Hospital, Valencia,
5 Instituto de Salud Carlos III,
6 Institute of Oncology,
7 Imperial College Healthcare National Health Service Trust, London,
8 Union Chimique Belge (UCB) Pharma, Monheim-am-Rhein,
9 UCB Pharma, Raleigh,
10 UCB Pharma, Slough,
11 PTx Solutions Ltd., London,
12 Weill Cornell Medicine, New York,
13 University Hospital of Wales, Cardiff
 
Disponibil în IBN: 15 martie 2021


Rezumat

Primary immune thrombocytopenia (ITP) is a predominantly immunoglobulin G (IgG)-autoantibody-mediated disease characterized by isolated thrombocytopenia. Rozanolixizumab, a subcutaneously infused humanized monoclonal anti-neonatal Fc receptor (FcRn) antibody, reduced serum IgG in healthy volunteers. In this phase 2, multicenter, open-label study, patients with persistent/chronic primary ITP received 1 to 5 once-weekly subcutaneous infusions of rozanolixizumab (cumulative doses, 15-21 mg/kg). Primary objectives were safety and tolerability, and secondary objectives were clinical efficacy (change in platelet count) and pharmacodynamic effect (change in IgG). In all, 51 (77.3%) of 66 patients reported 1 or more adverse events (AEs), all mild-to-moderate, most commonly headaches (26 [39.4%] of 66), of which 15 were treatment related. Four patients had serious AEs, but none were treatment related. No AEs resulted in discontinuation of the study drug. No serious infections occurred. Platelet counts of $50 3 109/L were achieved at least once at any time after multiple infusions (5 3 4, 3 3 7, or 2 3 10 mg/kg: 35.7%, 35.7%, and 45.5% of patients, respectively) or single infusions (15 or 20 mg/kg: 66.7% and 54.5% patients, respectively). Minimum mean IgG levels and maximum mean platelet counts both occurred by day 8 in the higher (15 and 20 mg/kg) single-dose cohorts and maximum platelet count occurred by day 11 onward in the multiple-dose cohorts. No clinically meaningful changes occurred in IgA, IgM, IgE, or albumin levels. In patients with persistent/chronic primary ITP, rozanolixizumab demonstrated a favorable safety profile and rapid, substantial platelet increases concordant with substantial IgG reductions, especially with single doses. By day 8, in the 15 and 20 mg/kg single-dose cohorts, .50% patients achieved clinically relevant platelet responses ($50 3 109/L), coinciding with the lowest mean IgG levels. These data support phase 3 development of rozanolixizumab in persistent/chronic primary ITP. 

Cuvinte-cheie
Idiopathic Thrombocytopenic Purpura, Romiplostim, Eltrombopag