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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
Who can join
Up to age 3 · All sexes
Full eligibility criteria
Inclusion Criteria: * 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 Criteria: Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).
About the study
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.
What is being tested
- Velmanase Alfa (drug)
Sponsor: Chiesi Farmaceutici S.p.A. · Participants: 5 · Started: Feb 19, 2025
Contact the study team
- Chiesi Clinical trials · Phone: +39.0521 2791
Official record on ClinicalTrials.gov — NCT06184503
Locations in the U.S.
| Florida | Nicklaus Children's Hospital, Miami |
| South Carolina | Greenwood Genetic Center, Greenwood |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.