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| Descriptive Information Fields | |||||
| Brief Title † | Long-Term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) | ||||
| Official Title † | A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose Study of the Efficacy and Long-Term Safety of Metazym (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy | ||||
| Brief Summary | 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). 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. The efficacy observed during the first 26 weeks of treatment in the phase I trial (EudraCT number: 2006-005341-11) will be studied further during totally 1 year of treatment. The myelin recovery (CNS and PNS) and functional capacity will be evaluated after this long-term treatment. |
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| Detailed Description | |||||
| Study Phase | Phase II | ||||
| Study Type † | Interventional | ||||
| Study Design † | Treatment, Non-Randomized, Open Label, Uncontrolled, Factorial Assignment, Efficacy Study | ||||
| Primary Outcome Measure † | Relative change in GMFM after 52 weeks of treatment Absolute change in Mullen's Scales of Early Learning, after 52 weeks of treatment. [ Time Frame: 52 weeks of tratment ] [ Designated as safety issue: No ] | ||||
| Secondary Outcome Measure † | Change in CSF biomarker after 52 weeks of treatment with focus on reduction of sulfatid. [ Time Frame: After 52 weeks of treatment ] [ Designated as safety issue: No ] | ||||
| Condition † | Late Infantile Metachromatic Leukodystrophy | ||||
| Intervention † | Drug: Metazym | ||||
| MEDLINE PMIDs | |||||
| Links | Sponsors homepage ![]() |
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| Recruitment Information Fields | |||||
| Recruitment Status † | Active, not recruiting | ||||
| Enrollment † | 12 | ||||
| Start Date † | February 2008 | ||||
| Completion Date | December 2008 | ||||
| Eligibility Criteria † | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||
| Ages | 1 Year to 5 Years | ||||
| Accepts Healthy Volunteers | No | ||||
| Contacts †† | |||||
| Location Countries † | Denmark | ||||
| Administrative Information Fields | |||||
| NCT ID † | NCT00633139 | ||||
| Organization ID | rhASA-03 | ||||
| Secondary IDs †† | rhASA-03, EudraCT number: 2007-006345-40 | ||||
| Study Sponsor † | Zymenex A/S | ||||
| Collaborators †† | |||||
| Investigators † |
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| Information Provided By | Zymenex A/S | ||||
| Verification Date | February 2008 | ||||
| First Received Date † | February 29, 2008 | ||||
| Last Updated Date | February 29, 2008 | ||||