drugset / Trial / NCT01411228
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Non-randomizedParallel-groupTriple-blindTreatment
Summary
A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to \<18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa).
Timeline
- Start
- 2011-09
- Primary completion
- 2014-07
- Completion
- 2014-08
Publications
- Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Cooper PA, Varughese S, Giraldo P, Petakov M, Tan ES, Chertkoff R. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naive or previously treated with imiglucerase. Blood Cells Mol Dis. 2018 Feb;68:163-172. doi: 10.1016/j.bcmd.2016.10.005. Epub 2016 Oct 20.
- Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Taliglucerase alfa | Protein / enzyme biologic | 30 iu/kg | Other |
| Subject | Taliglucerase alfa | Protein / enzyme biologic | 60 iu/kg | Other |