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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease
Blood, Volume 118, No. 22, Year 2011
Notification
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Description
Taliglucerase alfa (Protalix Biotherapeutics, Carmiel, Israel) is a novel plant cell-derived recombinant human β-glucocerebrosidase for Gaucher disease. A phase 3, double-blind, randomized, parallelgroup, comparison-dose (30 vs 60 U/kg body weight/infusion) multinational clinical trial was undertaken. Institutional review board approvals were received. A 9-month, 20-infusion trial used inclusion/exclusion criteria in treatmentnaive adult patients with splenomegaly and thrombocytopenia. Safety end points were drug-related adverse events: Ab formation and hypersensitivity reactions. Primary efficacy end point was reduction in splenic volume measured by magnetic resonance imaging. Secondary end points were: changes in hemoglobin, hepatic volume, and platelet counts. Exploratory parameters included biomarkers and bone imaging. Twenty-nine patients (11 centers) completed the protocol. There were no serious adverse events; drug-related adverse events were mild/moderate and transient. Two patients (6%) developed non-neutralizing IgG Abs; 2 other patients (6%) developed hypersensitivity reactions. Statistically significant spleen reductionwas achieved at 9 months: 26.9% (95% confidence interval [CI]: -31.9, -21.8) in the 30-unit dose group and 38.0% (95% CI: -43.4, -32.8) in the 60-unit dose group (both P < .0001); and in all secondary efficacy end point measures, except platelet counts at the lower dose. These results support safety and efficacy of taliglucerase alfa for Gaucher disease. This study was registered at www. clinicaltrials.gov as NCT00376168. © 2011 by The American Society of Hematology.
Authors & Co-Authors
Zimran, Ari
Israel, Jerusalem
Hebrew University of Jerusalem
Brill-Almon, Einat
Israel, Carmiel
Protalix Biotherapeutics
Chertkoff, Raul
Israel, Carmiel
Protalix Biotherapeutics
Petakov, Milan S.
Serbia, Belgrade
University of Belgrade
Blanco-Favela, Francisco
Mexico, Mexico
Instituto Mexicano Del Seguro Social
Muñoz, Eduardo Terreros
Mexico, Mexico
Instituto Mexicano Del Seguro Social
Solorio-Meza, Sergio E.
Mexico, Leon
Hospital de Especialidades No. 1
Amato, Dominick
Canada, Toronto
Mount Sinai Hospital of University of Toronto
Duran, Gloria
Chile, Santiago
Pontificia Universidad Católica de Chile
Giona, Fiorina
Italy, Rome
Sapienza Università Di Roma
Heitner, René
South Africa, Johannesburg
University of the Witwatersrand
Rosenbaum, Hanna
Israel, Haifa
Rambam Health Care Campus Israel
Giraldo, Pilar
Spain, Zaragoza
Hospital Universitario
Mehta, Atul B.
United Kingdom, London
The Royal Free Hospital
Park, Glen
United States, New York
Target Health Inc.
Phillips, Mici
Israel, Jerusalem
Hebrew University of Jerusalem
Elstein, Deborah
Israel, Jerusalem
Hebrew University of Jerusalem
Altarescu, Gheona
Israel, Jerusalem
Hebrew University of Jerusalem
Szleifer, Mali
Israel, Carmiel
Protalix Biotherapeutics
Hashmueli, Sharon
Israel, Carmiel
Protalix Biotherapeutics
Aviezer, David
Israel, Carmiel
Protalix Biotherapeutics
Statistics
Citations: 188
Authors: 21
Affiliations: 13
Identifiers
Doi:
10.1182/blood-2011-07-366955
ISSN:
00064971
Research Areas
Disability
Study Design
Exploratory Study