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

• Status: Completed
• Trial end date: 2020-07-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

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

Criteria

Inclusion Criteria:

1. Patient's custodial parent(s) must provide signed ICF prior to the involvement of the patient in any trial-related activities

2. The subject's custodial parent(s) must have the ability to comply with the protocol

3. 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)

4. The subject must have an age at the time of screening < 6 years.

Exclusion Criteria:

1. The subject's diagnosis cannot be confirmed by alpha-mannosidase activity < 10% of normal activity

2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis

3. History of BMT

4. 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

5. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial

6. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial

7. Participation in other interventional trials testing the IMP within the last 3 months.