title: A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin® in patients with type 1 Gaucher disease.
abstract: Gaucher disease is a lysosomal storage disease for which enzyme replacement therapy has proven to be effective. A switch-over clinical trial was performed to evaluate the efficacy and safety of Abcertin® (ISU Abxis, Seoul, Korea) in subjects with type 1 Gaucher disease who were previously treated with imiglucerase. Five Korean patients with type 1 Gaucher disease were enrolled. Previous doses of imiglucerase ranged from 30 to 55 U/kg every other week. The same dose of Abcertin® was administered to all patients for 24 weeks. Primary efficacy endpoints were changes in hemoglobin levels and platelet counts, and the secondary efficacy endpoints included changes in liver and spleen volumes, serum biomarkers, skeletal status and bone mineral density (BMD). During the study period, no statistically significant changes were observed in all parameters including hemoglobin levels and platelet counts, liver and spleen volumes, skeletal status and BMD. Abcertin® administration was continued in three patients for another 24 weeks as an extension of the study. Hemoglobin levels and platelet counts were maintained in all three patients. In conclusion, the efficacy and safety of Abcertin® are similar to those of imiglucerase, and Abcertin® is an effective therapeutic agent for patients with type 1 Gaucher disease (Clinical Trial Registry No. NCT02053896 at www.clinicaltrials.gov).
authors.affiliation: Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
authors.forename: Jin-Ho
authors.identifier: 0000-0003-1196-7826
authors.initials: JH
authors.lastname: Choi
authors.affiliation: Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
authors.forename: Beom Hee
authors.identifier: 0000-0001-9709-2631
authors.initials: BH
authors.lastname: Lee
authors.affiliation: Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Korea.
authors.forename: Jung Min
authors.identifier: 0000-0002-5752-6703
authors.initials: JM
authors.lastname: Ko
authors.affiliation: Department of Medical Genetics, Ajou University Hospital, Ajou University School of Medicine, Suwon, Korea.
authors.forename: Young Bae
authors.identifier: 0000-0002-4664-1941
authors.initials: YB
authors.lastname: Sohn
authors.affiliation: Department of Clinical Genetics, Yonsei University College of Medicine, Seoul, Korea.
authors.forename: Jin-Sung
authors.identifier: 0000-0002-1262-8597
authors.initials: JS
authors.lastname: Lee
authors.affiliation: Medical Genetics Center, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: Gu-Hwan
authors.identifier: 0000-0002-7056-7446
authors.initials: GH
authors.lastname: Kim
authors.affiliation: Medical Genetics Center, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: Sun Hee
authors.identifier: 0000-0002-5617-4582
authors.initials: SH
authors.lastname: Heo
authors.affiliation: ISU Abxis Co., Seongnam, Korea.
authors.forename: June-Young
authors.identifier: 0000-0001-8688-0907
authors.initials: JY
authors.lastname: Park
authors.affiliation: Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
authors.forename: Yoo-Mi
authors.identifier: 0000-0002-2922-8514
authors.initials: YM
authors.lastname: Kim
authors.affiliation: Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
authors.forename: Ja-Hye
authors.identifier: 0000-0001-8181-0694
authors.initials: JH
authors.lastname: Kim
authors.affiliation: Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
authors.forename: Han-Wook
authors.identifier: 0000-0001-8615-186X
authors.initials: HW
authors.lastname: Yoo
mesh_terms: D000293:Adolescent; D000328:Adult; D059451:Biosimilar Pharmaceuticals / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D002648:Child; D056947:Enzyme Replacement Therapy / Q000009:adverse effects; D005260:Female; D005776:Gaucher Disease / Q000097:blood / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D011994:Recombinant Proteins / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*
chemical_list: C000607357:Abcertin; D059451:Biosimilar Pharmaceuticals; D011994:Recombinant Proteins; D005962:Glucosylceramidase; C090568:imiglucerase
keywords: Enzyme Replacement Therapy; Gaucher Disease; Imiglucerase
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