drugset / Trial / NCT01303146
Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation
Phase 2
Completed
1 enrolled
Assistance Publique - Hôpitaux de Paris
European Leukodystrophy Association · collabShire · collabURC-CIC Paris Descartes Necker Cochin · collabZymenex A/S · collab
Non-randomizedSingle-groupOpen-labelTreatment
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).
Timeline
- Start
- 2008-10
- Primary completion
- 2010-03
- Completion
- 2010-04
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | rhARSA | Protein / enzyme biologic | 100 iu/kg | Intravenous |