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Sponsored by: |
Shire Human Genetic Therapies, Inc. |
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Information provided by: | Shire Human Genetic Therapies, Inc. |
ClinicalTrials.gov Identifier: | NCT00418561 |
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD.
Methodology: This is a single center, open-label study of patients with late infantile MLD. Twelve patients will be enrolled in this study receiving a total of thirteen intravenous infusions of Metazym. One infusion will be given every other week for a period of half a year. After the half year the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period.
Condition | Intervention | Phase |
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Metachromatic Leukodystrophy |
Drug: rhASA |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Factorial Assignment, Safety Study |
Official Title: | A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) |
Enrollment: | 12 |
Study Start Date: | January 2007 |
Study Completion Date: | March 2008 |
Primary Completion Date: | March 2008 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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rhASA: Experimental |
Drug: rhASA
intravenous infusion 25 U/kg,50 U/kg, 100U/kg or 200 U/kg every other week for 26 weeks
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Test product, dose, mode of administration, batch No.: The lowest dose level will be evaluated as a single dose of 25 U/kg. The three upper dose levels will be evaluated as repeated doses. Patients in each cohort will receive one dose of enzyme every other week for a period of eight weeks, a total of five doses. Dosing will be performed as follows: Cohort 1: 25 U/kg as a single dose - hereafter 50 U/kg; Cohort 2: 100 U/kg; Cohort 3: 200 U/kg. Patients receiving the lowest dose as a single dose will receive the next dose level as a repeated dose. After twenty six weeks the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period. The dose will be adjusted monthly to account for changes in body weight. The infusion length will be dependent on the dose. Doses of 25 U/kg, 50 U/kg and 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.
Duration of treatment: Half a year (26 weeks)
Ages Eligible for Study: | 1 Year to 5 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
The patient must have a confirmed diagnosis of MLD as defined by:
ASA activity < 10 nmol/h/mg in leukocytes Presence of elevated sulfatide in urine
Exclusion Criteria:
Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
Responsible Party: | Shire HGT ( Carol Cannon (U.S.) or Steve Moloney (EU) ) |
Study ID Numbers: | rhASA-01, EudraCT number: 2006-005341-11 |
Study First Received: | January 4, 2007 |
Last Updated: | December 3, 2008 |
ClinicalTrials.gov Identifier: | NCT00418561 |
Health Authority: | Denmark: Danish Medicines Agency |
Metazym MLD rhASA |
Lipid Metabolism, Inborn Errors Sphingolipidoses Leukodystrophy, Metachromatic Metabolic Diseases Demyelinating Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Demyelinating diseases Brain Diseases |
Leukodystrophy Metabolism, Inborn Errors Genetic Diseases, Inborn Metachromatic leukodystrophy Brain Diseases, Metabolic, Inborn Lipidoses Metabolic disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lysosomal Storage Diseases, Nervous System Nervous System Diseases Sulfatidosis Hereditary Central Nervous System Demyelinating Diseases |