J Korean Med Sci.  2015 Apr;30(4):378-384. 10.3346/jkms.2015.30.4.378.

A Phase 2 Multi-center, Open-label, Switch-over Trial to Evaluate the Safety and Efficacy of Abcertin(R) in Patients with Type 1 Gaucher Disease

• Choi JH
• Lee BH
• Ko JM
• Sohn YB
• Lee JS
• Kim GH
• Heo SH
• Park JY
• Kim YM
• Kim JH
• Yoo HW

• KMID: 2164444
• DOI: http://doi.org/10.3346/jkms.2015.30.4.378

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(R) (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(R) 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(R) 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(R) are similar to those of imiglucerase, and Abcertin(R) is an effective therapeutic agent for patients with type 1 Gaucher disease (Clinical Trial Registry No. NCT02053896 at www.clinicaltrials.gov).

Keyword

Gaucher Disease; Enzyme Replacement Therapy; Imiglucerase

MeSH Terms

Adolescent
Adult
Biosimilar Pharmaceuticals/adverse effects/pharmacokinetics/*therapeutic use
Child
*Enzyme Replacement Therapy/adverse effects
Female
Gaucher Disease/blood/*drug therapy
Glucosylceramidase/adverse effects/pharmacokinetics/*therapeutic use
Humans
Male
Recombinant Proteins/adverse effects/pharmacokinetics/*therapeutic use
Biosimilar Pharmaceuticals
Recombinant Proteins
Glucosylceramidase

Figure

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  Fig. 1 Mean change in hemoglobin levels (A), platelet count (B), angiotensin converting enzyme (C), acid phosphatase (D), chitotriosidase (E), and bone mineral density (BMD) of L-spine (F) and femur neck (G) in Abcertin® treated patients. The phase 2 clinical trial included five patients for 24th weeks. The study was extended by another 24 weeks for three subjects. Data are expressed as mean±standard deviation.

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