The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.
This protocol only concern subjects where compassionate use program was not accepted. Efficacy will be evaluated once yearly to follow the subject's progress in clinical parameters.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
ERT, i.v. infusions weekly
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, Denmark
Change from baseline in Adverse events
AE documented at all visits
Time frame: 3 year
Change from baseline in Lamazym antibodies
AB measured every 12th week
Time frame: 3 year
progress from baseline in number of steps climbed in 3 minutes
Time frame: 1 year, 2 year and 3 year
Progress from baseline in equivalent age
Time frame: 1 year, 2 year and 3 year
Progress from baseline in Forced Vital Capacity
Time frame: 1 year, 2 year and 3 year
Progress from baseline in distance walked in 6 minutes
Time frame: 1 year, 2 year and 3 year
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