The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
ERT, i.v. infusions weekly
Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
Brussels, Belgium
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, Denmark
Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
Córdoba, Spain
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
Manchester, United Kingdom
Reduction of Oligosaccharides in blood serum
Efficacy endpoint evaluation as change from baseline
Time frame: 6 months
The number of steps climbed in 3 minutes (3-minute stair climb)
Efficacy endpoint evaluation as change from baseline
Time frame: 6 months
Reduction of Oligosaccharides in CSF
Efficacy endpoint evaluation as change from baseline
Time frame: 6 months
The distance walked in 6 minutes (6-minute walk test)
Efficacy endpoint evaluation as change from baseline
Time frame: 6 months
Pulmonary function
Efficacy endpoint evaluation as change from baseline
Time frame: 6 months
Adverse events
Safety endpoint assesed weekly throughout the trial
Time frame: 1 week
Development of clinically significant changes in vital signs and change in physical examination
Safety endpoint assesed weekly throughout the trial
Time frame: 1 week
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
Safety endpoint assesed weekly throughout the trial
Time frame: 1 week
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
Safety endpoint assesed weekly throughout the trial
Time frame: 1 week