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

Primary Purpose

Late Infantile Metachromatic Leukodystrophy

Status
Terminated
Phase
Phase 2
Locations
Denmark
Study Type
Interventional
Intervention
HGT-1111
Sponsored by
Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Late Infantile Metachromatic Leukodystrophy focused on measuring Metazyme, Late Infantile Metachromatic Leukodystrophy

Eligibility Criteria

3 Years - 6 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Rigshospitalet

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

HGT-1111 100 U/kg

HGT-1111 200 U/kg

Arm Description

Outcomes

Primary Outcome Measures

Days of Exposure to HGT-1111
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.

Secondary Outcome Measures

Level of Cerebrospinal Fluid (CSF) Sulfatide
Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
Level of White Matter Metabolites
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)
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.

Full Information

First Posted
May 19, 2008
Last Updated
May 19, 2021
Sponsor
Shire
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1. Study Identification

Unique Protocol Identification Number
NCT00681811
Brief Title
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Terminated
Why Stopped
Terminated prior to planned completion date due to lack of efficacy.
Study Start Date
February 20, 2008 (Actual)
Primary Completion Date
October 22, 2010 (Actual)
Study Completion Date
October 22, 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire

4. Oversight

Data Monitoring Committee
No

5. Study Description

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 Description
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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Late Infantile Metachromatic Leukodystrophy
Keywords
Metazyme, Late Infantile Metachromatic Leukodystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
HGT-1111 100 U/kg
Arm Type
Experimental
Arm Title
HGT-1111 200 U/kg
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
HGT-1111
Other Intervention Name(s)
rhASA, Metazym
Intervention Description
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.
Primary Outcome Measure Information:
Title
Days of Exposure to HGT-1111
Description
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.
Time Frame
Baseline until end of study (Week 139)
Secondary Outcome Measure Information:
Title
Level of Cerebrospinal Fluid (CSF) Sulfatide
Description
Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
Time Frame
Baseline until end of study (Week 139)
Title
Level of White Matter Metabolites
Description
Level of white matter metabolites [N-acetyl Aspartate (NAA)] measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
Time Frame
Baseline until end of study (Week 139)
Title
Score of Gross Motor Function Measurement (GMFM)
Description
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.
Time Frame
Baseline until end of study (Week 139)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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) Completion of study HGT-MLD-048 (NCT00633139) The subject and his/her guardian(s) must have the ability to comply with the protocol Exclusion Criteria: Spasticity so severe to inhibit transportation 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 Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Rigshospitalet
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark

12. IPD Sharing Statement

Citations:
PubMed Identifier
33332761
Citation
I Dali C, Groeschel S, Moldovan M, Farah MH, Krageloh-Mann I, Wasilewski M, Li J, Barton N, Krarup C. Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. Ann Clin Transl Neurol. 2021 Jan;8(1):66-80. doi: 10.1002/acn3.51254. Epub 2020 Dec 17.
Results Reference
derived

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

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