Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation
Status:
Completed
Trial end date:
2010-04-01
Target enrollment:
Participant gender:
Summary
There is currently no effective treatment for late infantile MLD once clinical symptoms are
evident. METAZYM is a recombinant human arylsulfatase A developed for an intravenous ERT for
the treatment of late infantile MLD. The overall objective of this study is to evaluate the
efficacy and safety of intravenous rhASA treatment in a patient with late infantile MLD who
had previously received hematopoietic stem cell transplantation (HCT).
Phase:
Phase 2
Details
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborators:
European Leukodystrophy Association Shire Zymenex A/S