Status:
RECRUITING
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
Lead Sponsor:
Chiesi Farmaceutici S.p.A.
Conditions:
Alpha-Mannosidosis
Eligibility:
All Genders
Up to 3 years
Brief 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 ...
Eligibility Criteria
Inclusion
- 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
- Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).
Key Trial Info
Start Date :
February 19 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 1 2029
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT06184503
Start Date
February 19 2025
End Date
September 1 2029
Last Update
May 16 2025
Active Locations (2)
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1
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
2
Greenwood Genetic Center
Greenwood, South Carolina, United States, 29605