Status:
WITHDRAWN
Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis
Lead Sponsor:
Chiesi Farmaceutici S.p.A.
Conditions:
Alpha-Mannosidosis
Eligibility:
All Genders
Phase:
PHASE3
Brief 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 cl...
Detailed Description
A Screening visit (V1) will take place 7±3 days prior to randomization in order to give the subject enough time to consider their participation in the study, to plan the next visits including the long...
Eligibility Criteria
Inclusion
- 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
- 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).
Key Trial Info
Start Date :
January 11 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 29 2021
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04031066
Start Date
January 11 2021
End Date
December 29 2021
Last Update
December 8 2020
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