Trial intelligence / Profile preview

Natural course and enzyme replacement therapy in mucopolysaccharidosis I, II, VI

2007-001453-26Phase 4Active

Start
Nov 2009
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
Countries
1
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Overview

Phase 4, open-label trial of laronidase, idursulfase and galsulfase in mucopolysaccharidosis type I, mucopolysaccharidosis type 2, mucopolysaccharidosis type VI and 1 more (2 arms). Primary endpoint: Survival. Started Nov 2009; status: active.

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Drugs

laronidaseidursulfasegalsulfase
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Conditions

mucopolysaccharidosis type Imucopolysaccharidosis type 2mucopolysaccharidosis type VIMorquio syndrome

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