- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00681811
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
May 19, 2021 updated by: Shire
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.
Study Overview
Status
Terminated
Conditions
Intervention / Treatment
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).
Study Type
Interventional
Enrollment (Actual)
11
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Copenhagen, Denmark, 2100
- Rigshospitalet
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
3 years to 6 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: HGT-1111 100 U/kg
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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 Names:
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Experimental: HGT-1111 200 U/kg
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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 Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Days of Exposure to HGT-1111
Time Frame: Baseline until end of study (Week 139)
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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.
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Baseline until end of study (Week 139)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Level of Cerebrospinal Fluid (CSF) Sulfatide
Time Frame: Baseline until end of study (Week 139)
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Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
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Baseline until end of study (Week 139)
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Level of White Matter Metabolites
Time Frame: Baseline until end of study (Week 139)
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Level of white matter metabolites [N-acetyl Aspartate (NAA)] measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
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Baseline until end of study (Week 139)
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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.
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Baseline until end of study (Week 139)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
February 20, 2008
Primary Completion (Actual)
October 22, 2010
Study Completion (Actual)
October 22, 2010
Study Registration Dates
First Submitted
May 19, 2008
First Submitted That Met QC Criteria
May 19, 2008
First Posted (Estimate)
May 21, 2008
Study Record Updates
Last Update Posted (Actual)
June 8, 2021
Last Update Submitted That Met QC Criteria
May 19, 2021
Last Verified
May 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Demyelinating Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Leukoencephalopathies
- Hereditary Central Nervous System Demyelinating Diseases
- Sulfatidosis
- Leukodystrophy, Metachromatic
Other Study ID Numbers
- HGT-MLD-049
- 2008-000084-41 (EudraCT Number)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Late Infantile Metachromatic Leukodystrophy
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ShireCompletedLate Infantile Metachromatic LeukodystrophyDenmark
-
Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy AssociationCompletedLate Infantile Metachromatic LeukodystrophyFrance
-
ShireCompletedMetachromatic Leukodystrophy (MLD)Australia, Denmark, Japan, France, Germany
-
ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
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Shenzhen Geno-Immune Medical InstituteUnknown
-
TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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ShireCompletedMetachromatic Leukodystrophy (MLD)Denmark
-
ShireTerminatedMetabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditionsUnited States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
-
The Cooper Health SystemCompletedMetachromatic LeukodystrophyUnited States
Clinical Trials on HGT-1111
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Assistance Publique - Hôpitaux de ParisShire; Zymenex A/S; European Leukodystrophy AssociationCompletedMetachromatic LeukodystrophyFrance
-
ShireCompletedLate Infantile Metachromatic LeukodystrophyDenmark
-
ShireCompletedMetachromatic Leukodystrophy (MLD)Denmark
-
ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
-
TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
-
Vir Biotechnology, Inc.Bill and Melinda Gates FoundationCompletedHIV I InfectionUnited States
-
ShireCompletedMetachromatic Leukodystrophy (MLD)Australia, Denmark, Japan, France, Germany
-
ShireActive, not recruitingHunter SyndromeUnited States, Spain, Canada, United Kingdom, Mexico, Australia, France
-
ShireTerminatedSanfilippo Syndrome | Mucopolysaccharidosis (MPS)United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
-
ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy