Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis (SHAMAN)

This is an interventional treatment trial for Alpha-Mannosidosis focused on measuring Alpha-Mannosidosis, velmanase alfa, alpha-mann, shaman, mannosidosis, alpha-mannosidasis Read More

Inclusion Criteria:

• Confirmed diagnosis of alpha-mannosidosis based on alpha mannosidase activity <10% of normal in leukocytes or fibroblasts or through genetic testing;
• Capability to comply with the protocol;
• Evidence of informed consent provided by subject or legally authorized guardian(s) prior to performance of any trial-related activities.

Exclusion Criteria:

• Previous hematopoietic stem cells transplantation (HSCT) with positive outcome;
• Major surgery planned within 3 months prior to study entry or planned during the study that, in the opinion of the Investigator, would preclude participation in the trial;
• Known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that would preclude participation in the study in the Investigator's judgment;
• Pregnant (as evident by a positive urine hCG or serum-hCG test) or lactating women and all women physiologically capable of becoming pregnant (i.e. women of childbearing potential [WOCBP]) UNLESS they are willing to use highly effective birth control methods;
• Participation in other interventional trials testing investigational medicinal products (IMPs) within the last 6 months;
• Total IgE >800 IU/ml;
• Any hypersensitivity to velmanase alfa or its excipients that, in the judgment of the Investigator, places the subject at an increased risk for adverse reactions
• Clinically active infection and recent vaccinations (within the last month before screening).