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

Study Overview
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Brief Summary
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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.

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

Conditions
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Alpha-Mannosidosis

Intervention / Treatment
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* Drug: Velmanase Alfa


Participation Criteria
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Eligibility Criteria
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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).

Ages Eligible for Study
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Maximum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age<br>Paediatric patients with a confirmed diagnosis of alpha-mannosidosis with data for at least one pre- and one post-Lamzede treatment sample obtained when < 3 YOA. | Drug: Velmanase Alfa<br>* 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.<br>* Other names: Lamzede;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacodynamic Response to velmanase alfa | Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood | 52 weeks of treatment |