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

• Status: Withdrawn
• Trial end date: 2021-12-29
• Target enrollment: 0
• Participant gender: All

Summary

Randomized, double-blind, placebo-controlled, parallel group study where subjects will receive velmanase alfa or placebo for 24 weeks.

Each subject undergoes to 8 complete visits at the clinic for clinical, laboratory and functional assessments. Study treatment is administered weekly through i.v. infusions

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Chiesi Farmaceutici S.p.A.

Criteria

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).