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

Study Identifier:
CLI-LMZYMAA2-01
ClinicalTrials.gov Identifier:
NCT04031066
EudraCT Identifier:
N/A
EU CT ID:
N/A
Study Contact Information:
N/A
Terminated/Withdrawn

Study Details

Medical Condition
  • Alpha-mannosidosis
Study Drug
  • Drug: Velmanase Alfa
  • Drug: Placebo
Date
Jan 2021 - Oct 2021
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion 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).
Healthy Volunteers
No

Protocol 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

Study Locations

No locations found.