Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Summary
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.
Timeline
- Start
- 2007-01-22
- Primary completion
- 2008-03-27
- Completion
- 2008-03-27
Outcome
Missed primary endpoint
registry analysis (unspecified test); Cohort 1 vs Cohort 2 vs Cohort 3; p = 0.0737; ANOVA; note: Test for no difference between cohorts. NCT00418561 ↗
registry analysis (unspecified test); Cohort 1 vs Cohort 2 vs Cohort 3; p = 0.1115; ANOVA; note: Test for no difference between cohorts. NCT00418561 ↗
registry analysis (unspecified test); Cohort 1 vs Cohort 2 vs Cohort 3; p = 0.1268; ANOVA; note: Test for no difference between cohorts. NCT00418561 ↗
paper Motor function and developmental testing scores declined during the study in all dose groups; no significant differences were observed between groups. PMID 33332761 ↗
Publications
- Results Dali CI, Barton NW, Farah MH, Moldovan M, Mansson JE, Nair N, Duno M, Risom L, Cao H, Pan L, Sellos-Moura M, Corse AM, Krarup C. Sulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy. Ann Clin Transl Neurol. 2015 May;2(5):518-33. doi: 10.1002/acn3.193. Epub 2015 Mar 27.
- 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.