Clinical Trial: Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients With Late Infantile Metachr

Brief Summary: 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.

Detailed Summary: 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).
Sponsor: Shire

Current Primary Outcome: Days of Exposure to HGT-1111 [ Time Frame: Baseline until end of study (Week 139) ]

End of study was defined as until HGT-1111 was commercially available, the participant's participation was discontinued, or the study was terminated by the Sponsor.


Original Primary Outcome: The objective is to supply patients who have participated in the clinical development program of Metazym with treatment. [ Time Frame: Until the product is available for sale, market or the project is terminated ]

Current Secondary Outcome:

  • Level of Cerebrospinal Fluid (CSF) Sulfatide [ Time Frame: Baseline until end of study (Week 139) ]
    Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
  • Level of White Matter Metabolites [ Time Frame: Baseline until end of study (Week 139) ]
    Level of white matter metabolites [N-acetyl Aspartate (NAA)] measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
  • Score of Gross Motor Function Measurement (GMFM) [ Time Frame: Baseline until end of study (Week 139) ]
    Gross motor function was measured using GMFM-88 at 6-month intervals. The GMFM-88 item scores were summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) to 264 (maximum). Decrease in GMFM score indicates disease progression.


Original Secondary Outcome: The secondary objective of this study objective is ongoing evaluation of disease progress and safety profile of Metazym treatment in patients with late infantile MLD. [ Time Frame: Until Metazym is available for sale, market or the project is terminated ]

Information By: Shire

Dates:
Date Received: May 19, 2008
Date Started: February 2008
Date Completion:
Last Updated: August 4, 2015
Last Verified: February 2014