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
Chiesi Farmaceutici S.p.A.
5 participants
Feb 19, 2025
OBSERVATIONAL
Conditions
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.
Eligibility
Inclusion Criteria6
- Subjects with the provision of informed consent from their legal guardians (LAR)
- Have a confirmed diagnosis of alpha mannosidosis
- Have initiated treatment with velmanase alfa between birth to at least six weeks before turning 3 years of age
- Have information on the disease marker GlcNAc(Man)2 obtained:
- before velmanase treatment initiation (ideally max 6 month before), 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 Criteria1
- Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).
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Interventions
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.
Locations(2)
View Full Details on ClinicalTrials.gov
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NCT06184503