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

CLI-LMZYMAA2-04

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.

2024-05-01

2029-09-01

2029-09-01

Not yet recruiting

5

Inclusion Criteria: * Participants with the provision of informed consent from their legal guardians * Confirmed diagnosis of alpha mannosidosis * Velmanase alfa treatment initiation from birth to at least six weeks before turning 3 YOA, where the standard of care follow-up includes the following: * Data for GlcNAc(Man)2 levels obtained when the participant was \< 3 YOA for: * At least one pre-treatment sample obtained no more than 13 weeks before initiating velmanase alfa treatment, and * At least one post-treatment sample, collected following at least six weeks of treatment. * Participants treated with Lamzede, 1 mg/kg body weight, via weekly intravenous infusions. Exclusion Criteria: Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'OTHER', 'timePerspective': 'OTHER'}, 'enrollmentInfo': {'count': 5, 'type': 'ESTIMATED'}}

2023-12-28