Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Safety and tolerability of velmanase alfa as per Adverse events |
Safety and tolerability assessed as per AEs including infusion-related reactions [IRRs] |
From baseline throughout study completion, at least of 2 years |
|
Primary |
Safety and tolerability of velmanase alfa as per vital signs |
|
From baseline throughout study completion, at least of 2 years |
|
Primary |
Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per hematology |
|
From baseline throughout study completion, at least of 2 years |
|
Primary |
Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per blood biochemistry |
|
From baseline throughout study completion, at least of 2 years |
|
Primary |
Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per urinalysis |
|
From baseline throughout study completion, at least of 2 years |
|
Primary |
Detection of anti-velmanase alfa-IgG antibodies (ADA) and neutralizing/inhibitory antibodies |
Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained |
From baseline throughout study completion, at least of 2 years |
|
Secondary |
Evaluation of levels of Serum oligosaccharides |
Assessment of change from baseline in levels of Serum oligosaccharides |
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Functional capacity: The Peabody Developmental Motor Scale test (PDMS-2) |
Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained |
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Functional capacity: Bruininks-Oseretsky test of Motor Proficiency (BOT-2) when applicable by age (from 4 years) or upon the judgment of the physician |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Functional capacity: Mullen Scales of Early Learning (MSEL) |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Endurance: 3-Minute Stair Climb Test (3MSCT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Endurance: 6-Minute Walk Test (6MWT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician 2-Minute Walk Test (2MWT) in pediatric patients below 4 years of age |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Hearing evaluation: Otoacoustic Emissions (OAE) testing |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Hearing evaluation: Automatic Auditory Brainstem Response (A-ABR) audiometry |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Immunological profile when applicable upon the judgement of the physician (Serum IgG, IgA, IgM; in vitro synthesis of IgG; in vitro proliferative response and Immunophenotype) |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
CSF biomarkers: Tau protein (Tau) § Neurofilament Protein Light (NFL) § Glial Fibrillary Acidic Protein (GFAp) § Oligosaccharides |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Assessment of quality of life via Questionnaire |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Assessment of mannose-rich oligosaccharides in brain tissue, as measured by Magnetic Resonance Spectroscopy (MRS) |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Magnetic Resonance Imaging (MRI) in white matter, gray matter and in centrum semi ovale, and diffusion-MRI of the brain, |
|
From baseline throughout study completion, at least for 2 years |
|
Secondary |
Pharmacokinetic parameters to determine Cmax (Peak Concentration) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine Ctrough (Trough Plasma Concentration) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine Area Under Curve (AUC24) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine AUClast (Area Under Curve After The Last Count) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine AUCinf (Area Under Curve From Time Zero To Infinity) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine tmax (Time To Peak Concentration) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine CL (Clearance) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine t1/2 (Elimination Half-Life) |
|
At first dose (visit 1) and after 6 months (visit 26) |
|
Secondary |
Pharmacokinetic parameters to determine Rac (Obs) Observed Accumulation Ratio |
|
At first dose (visit 1) and after 6 months (visit 26) |
|