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Imiglucerase dose escalation versus imiglucerase + miglustat in type I Gaucher disease with active bone disease
2007-005516-61Phase 4Completed
- Sponsor
- Istituto Giannina Gaslini
- Start
- Mar 2009
- Modality
- Small Molecules, Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 4, randomised trial in Gaucher disease type 1 (GD1) comparing High-dose imiglucerase with Imiglucerase + miglustat (2 arms). Primary endpoint: Primary efficacy and safety assessment. Started Mar 2009; status: completed.
02
Drugs
imiglucerasemiglustat
03
Conditions
Gaucher disease type 1 (GD1)
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