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

What this Trial Is About

Researchers are studying the effects of the drug velmanase alfa (Lamzede®) in children under 3 years old who have Alpha-Mannosidosis, a rare genetic condition. The study aims to understand how velmanase alfa affects a specific disease marker called GlcNAc(Man)2 after one year of treatment and to observe how the child's body responds to the drug during therapy. This research collects data from real-world treatment experiences, including routine clinical tests and other observational studies. Participants are pediatric patients diagnosed with alpha-mannosidosis who have started treatment with velmanase alfa before turning three years old. They receive Lamzede® as an intravenous enzyme replacement therapy at a dose of 1 mg/kg once weekly. The study uses existing clinical data collected before and after treatment, focusing on samples taken prior to treatment initiation and after at least six weeks of therapy. During the study, parents or legal guardians provide test results from routine healthcare visits related to their child's general health and the administration of velmanase alfa. Researchers analyze these data to measure pharmacodynamic response over 52 weeks of treatment, focusing on changes in the disease marker GlcNAc(Man)2. The study continues until September 2029 and does not involve additional interventions beyond standard care and data collection.

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