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.
10.3346/jkms.2015.30.4.378
- Author:
Jin Ho CHOI
1
;
Beom Hee LEE
;
Jung Min KO
;
Young Bae SOHN
;
Jin Sung LEE
;
Gu Hwan KIM
;
Sun Hee HEO
;
June Young PARK
;
Yoo Mi KIM
;
Ja Hye KIM
;
Han Wook YOO
Author Information
1. Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea. hwyoo@amc.seoul.kr
- Publication Type:Clinical Trial, Phase II ; Original Article ; Multicenter Study ; Research Support, Non-U.S. Gov't
- Keywords:
Gaucher Disease;
Enzyme Replacement Therapy;
Imiglucerase
- MeSH:
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
- From:Journal of Korean Medical Science
2015;30(4):378-384
- CountryRepublic of Korea
- Language:English
-
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).
