NCT06184503

Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3

A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 5 (estimated)
• Sponsor: Chiesi Farmaceutici S.p.A. · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis. The main questions it aims to answer are: * study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy * explore how the child's body reacts to velmanase alfa during the therapy The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa. Additional data will be extracted from other observational sponsored studies/registries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing.

Conditions

• Alpha-Mannosidosis

Interventions

Type	Name	Description
DRUG	Velmanase Alfa	Lamzede® (velmanase alfa, henceforth referred to as Lamzede) is a recombinant human lysosomal alpha-mannosidase product developed as an intravenous enzyme replacement therapy (ERT) for the treatment of alpha-mannosidosis.

Timeline

• Start date: 2025-02-19
• Primary completion: 2029-09-01
• Completion: 2029-09-01
• First posted: 2023-12-28
• Last updated: 2025-05-16

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study