- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00633139
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
May 19, 2021 updated by: Shire
A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD.
All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
All patients will be offered continuing treatment in this study and will in this protocol receive 13 infusions, whereby the patients total have had 27 infusions of Metazym.
One infusion will be given every other week.
After a total of 52 weeks of treatment the subjects will continue treatment in a compassionate use protocol.
Safety (AE/SAE) will be monitored at every visit.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
13
Phase
- Phase 2
- Phase 1
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
-
-
-
Hvidovre, Denmark, 2650
- PhaseOneTrials A/S
-
-
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
1 year to 5 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
The patients from the Phase I trial must meet the following criteria to be enrolled in the study.
- 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)
- The subject and his/her guardian(s) must have the ability to comply with the clinical 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
- Use of any investigational product other than rhASA within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations
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: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Cohort 1
Cohort 1: 50 U/kg Recombinant human Arylsulfatase A (rhASA)
|
intravenous infusion, every other week for 26 weeks
Other Names:
|
Experimental: Cohort 2
Cohort 2: 100 U/kg Recombinant human Arylsulfatase A (rhASA)
|
intravenous infusion, every other week for 26 weeks
Other Names:
|
Experimental: Cohort 3
Cohort 3: 200 U/kg Recombinant human Arylsulfatase A (rhASA)
|
intravenous infusion, every other week for 26 weeks
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Relative Changes (%) in Gross Motor Function Measurement (GMFM)
Time Frame: Baseline, 52 Weeks
|
Change (percent change) in GMFM is measured from baseline to end of study (Week 52).
GMFM is measured using GMFM-88.
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).
Relative changes in GMFM are calculated as percentage change from baseline divided by the age difference in months between first and last visit.
The GMFM score decreases over time, which, indicates that the disease worsened over time.
Score over time (SOT), data mentioned over mean represents the adjusted mean.
|
Baseline, 52 Weeks
|
Relative Change in Mullen's Scales of Early Learning
Time Frame: Baseline, 52 Weeks
|
Changes in Mullen's Scales of Early Learning are measured from baseline to end of study (Week 52) using Mullen's Scales of Early Learning.
T scores, percentile ranks, and age equivalents can be computed for the four scales separately (visual reception, fine motor, expressive language, and receptive language).
Relative change is calculated as percentage change from baseline divided by the age-difference in months between first and last visit.
When Mullen's score decreases over time, it indicates the disease worsened over time.
Data mentioned over mean represents the adjusted mean.
|
Baseline, 52 Weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Cerebrospinal Fluid (CSF) Sulfatide
Time Frame: Baseline, 52 Weeks
|
Changes in CSF sulfatide from baseline to end of study (Week 52).
Data mentioned over mean represents the adjusted mean.
|
Baseline, 52 Weeks
|
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)
January 22, 2007
Primary Completion (Actual)
November 25, 2008
Study Completion (Actual)
November 25, 2008
Study Registration Dates
First Submitted
February 29, 2008
First Submitted That Met QC Criteria
February 29, 2008
First Posted (Estimate)
March 11, 2008
Study Record Updates
Last Update Posted (Actual)
June 14, 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-048
- 2007-006345-40 (EudraCT Number)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
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
-
ShireTerminatedLate 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
-
Shenzhen Geno-Immune Medical InstituteUnknown
-
TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
-
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
-
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 Recombinant human Arylsulfatase A (rhASA)
-
ShireCompletedMetachromatic Leukodystrophy (MLD)Australia, Denmark, Japan, France, Germany
-
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
-
ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
-
BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI | Maroteaux-Lamy SyndromeUnited States, Portugal, France
-
Bio Sidus SARecruiting
-
Green Cross CorporationICON Clinical ResearchTerminated
-
ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Denmark, Belgium, United Kingdom, Norway, Italy, Czechia
-
ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Serbia, Spain, United Kingdom, Australia, Paraguay
-
ProtalixWithdrawn
-
Greenovation Biotech GmbHFGK Clinical Research GmbHCompleted