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
@article{Choi2015AP2, 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}, author={J. Choi and B. Lee and J. M. Ko and Y. B. Sohn and Jin-Sung Lee and G. Kim and S. Heo and June-Young Park and Y. Kim and Ja-Hye Kim and Han-Wook Yoo}, journal={Journal of Korean Medical Science}, year={2015}, volume={30}, pages={378 - 384} }
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… CONTINUE READING
Supplemental Clinical Trials
Observational Clinical Trial
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with
Type 1 Gaucher disease previously treated with Imiglucerase.
Conditions | Gaucher Disease |
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8 Citations
Substrate reduction therapy as a new treatment option for patients with Gaucher disease type 1: A review of literatures
- Medicine
- 2016
- PDF
The road to biosimilars in rare diseases ‐ ongoing lessons from Gaucher disease
- Medicine
- American journal of hematology
- 2019
Recent advances and future challenges in Gaucher disease.
- Medicine
- Blood cells, molecules & diseases
- 2018
- 6
References
SHOWING 1-10 OF 27 REFERENCES
Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study
- Medicine
- American journal of hematology
- 2013
- 50
Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience.
- Medicine
- Blood
- 2010
- 106
Comparative Therapeutic Effects of Velaglucerase Alfa and Imiglucerase in a Gaucher Disease Mouse Model
- Medicine, Chemistry
- PloS one
- 2010
- 37
- PDF
Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.
- Medicine
- Blood cells, molecules & diseases
- 2010
- 80
- PDF
A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease.
- Medicine
- Blood cells, molecules & diseases
- 2007
- 52
The long-term international safety experience of imiglucerase therapy for Gaucher disease.
- Medicine
- Molecular genetics and metabolism
- 2007
- 106
The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review.
- Medicine
- Health technology assessment
- 2006
- 85
- PDF
Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease.
- Medicine
- The New England journal of medicine
- 1991
- 1,081