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Phase 2/3 randomized double-blind placebo-controlled study of olipudase alfa in acid sphingomyelinase deficiency (Niemann-Pick disease type B)
2015-000371-26Phase 2Completed
- Sponsor
- Sanofi
- Start
- Jun 2015
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial in acid sphingomyelinase deficiency (ASMD) and Niemann-Pick disease type B (NPD-B) comparing Olipudase alfa with Placebo (2 arms). Primary endpoint: Percentage change in spleen volume. Started Jun 2015; status: completed.
02
Drugs
olipudase alfa
03
Conditions
acid sphingomyelinase deficiency (ASMD)Niemann-Pick disease type B (NPD-B)
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