Late Infantile Metachromatic Leukodystrophy Clinical Trial
Official title:
A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures. This group of patients will be offered ongoing treatment with HGT-1111 in this protocol. One infusion will be given every other week until the product is commercially available, the patient discontinues, or the study is terminated by the Sponsor, provided no safety issues have emerged.
The primary objective of this study is to provide ongoing treatment of HGT-1111 to patients who have completed study HGT-MLD-048 (previously study rhASA-03 - NCT00633139) until HGT-1111 is commercially available or the study is terminated by the Sponsor, provided no safety concerns have emerged. The secondary objective of this study is to monitor disease progression and the safety profile of HGT-1111 administered to patients who have completed study HGT-MLD-048 (NCT00633139). ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT01325025 -
Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy
|
N/A | |
| Completed |
NCT00633139 -
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
|
Phase 1/Phase 2 |