Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis

Study Identifier:
CCD-LMZYMAA1-08
ClinicalTrials.gov Identifier:
NCT02998879
EudraCT Identifier:
2016-001988-36
EU CT ID:
N/A
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Alpha-mannosidosis
Study Drug
  • Drug: Velmanase Alfa (e.g. Lamazym)
Date
Dec 2016 - Jul 2020
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 6 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Patient's custodial parent(s) must provide signed ICF prior to the involvement of the patient in any trial-related activities
  • The subject's custodial parent(s) must have the ability to comply with the protocol
  • The subject must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity in leukocytes or fibroblasts \< 10% of normal activity (historical data)
  • The subject must have an age at the time of screening \< 6 years.
Exclusion Criteria
  • The subject's diagnosis cannot be confirmed by alpha-mannosidase activity \< 10% of normal activity
  • Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
  • History of BMT
  • Presence of known clinically significant cardiovascular, hepatic, pulmonary, or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
  • Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
  • Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
  • Participation in other interventional trials testing the IMP within the last 3 months.
Healthy Volunteers
No

Protocol Summary

The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years

Study Locations

Location
Status
Location
Vienna, Austria
Status
N/A
Location
Copenhagen, Denmark
Status
N/A
Location
Lyon, France
Status
N/A
Location
Hamburg, Germany
Status
N/A
Location
Mainz, Germany
Status
N/A
Location
Trieste, Italy
Status
N/A