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

This study has been terminated.
(Terminated prior to planned completion date due to lack of efficacy.)
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00681811
First received: May 19, 2008
Last updated: February 19, 2014
Last verified: February 2014

May 19, 2008
February 19, 2014
February 2008
October 2010   (final data collection date for primary outcome measure)
Days of Exposure to HGT-1111 [ Time Frame: Baseline until end of study ] [ Designated as safety issue: No ]
End of study defined as until HGT-1111 is commercially available, the patient's participation is discontinued, or the study is terminated by the Sponsor
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 ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00681811 on ClinicalTrials.gov Archive Site
  • Level of Cerebrospinal Fluid (CSF) Sulfatide [ Time Frame: Baseline until end of study ] [ Designated as safety issue: No ]
    Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049
  • Level of White Matter Metabolites [ Time Frame: Baseline until end of study ] [ Designated as safety issue: No ]
    Level of white matter metabolites [N-acetyl Aspartate (NAA)] measured at 6-month intervals in HGT-MLD-049
  • Score of Gross Motor Function Measurement (GMFM) [ Time Frame: Baseline until end of study ] [ Designated as safety issue: No ]
    Gross motor function is measured using GMFM-88 and measured at 6-month intervals in HGT-MLD-049 study. The GMFM-88 item scores can be summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score is between 0 (minimal) to 3 (maximum). The total GMFM-88 score is between 0 (minimal) to 264 (maximum). With GMFM score decreases over time, it indicates disease progression.
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 ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
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).

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Late Infantile Metachromatic Leukodystrophy
Drug: HGT-1111
Patients currently dosed with 100 U/kg or 200 U/kg will continue this treatment. Patients dosed with 50 U/kg will be equally randomized to treatment on 100 U/kg or 200 U/kg. The dose will be adjusted every 6-week to account for changes in body weight.The infusion length will be dependent on the dose. Infusion of 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes.
Other Name: rhASA, Metazym
  • Experimental: HGT-1111 100 U/kg
    Intervention: Drug: HGT-1111
  • Experimental: HGT-1111 200 U/kg
    Intervention: Drug: HGT-1111
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
11
October 2010
October 2010   (final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (Trial-related activities are any procedures that would not have been performed during normal management of the subject)
  2. Completion of study HGT-MLD-048 (NCT00633139)
  3. The subject and his/her guardian(s) must have the ability to comply with the protocol

Exclusion Criteria:

  1. Spasticity so severe to inhibit transportation
  2. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that, in the opinion of the Investigator, would preclude participation in the trial
  3. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
Both
3 Years to 6 Years
No
Contact information is only displayed when the study is recruiting subjects
Denmark
 
NCT00681811
HGT-MLD-049, 2008-000084-41
No
Shire
Shire
Not Provided
Principal Investigator: Christine Dali, MD Rigshospitalet, Denmark
Shire
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP