Actively Recruiting
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
Led by Chiesi Farmaceutici S.p.A. · Updated on 2025-05-16
5
Participants Needed
2
Research Sites
236 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Legal guardian provides informed consent
- Confirmed diagnosis of alpha mannosidosis
- Started velmanase alfa treatment between birth and before 3 years old
- Has disease marker GlcNAc(Man)2 measured before treatment (within 6 months) and after at least 6 weeks of treatment
- Received velmanase alfa at 1 mg/kg via weekly intravenous infusions
You will not qualify if you...
- Prior hematopoietic stem cell transplantation (HSCT)
- Use of other investigational treatments for alpha mannosidosis (supportive treatments allowed)
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
2
Greenwood Genetic Center
Greenwood, South Carolina, United States, 29605
Actively Recruiting
Research Team
C
Chiesi Clinical trials
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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