Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
Recruiting
Observational Study
Alpha-Mannosidosis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- Up to 3
- Sex
- Any
- Study type
- Observational
- Participants needed
- 5 (estimated)
- Sponsor
- Chiesi Farmaceutici S.p.A. · Industry
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
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 Th…
Trial Locations
Loading…
Loading trial locations…
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 diseas…
Contacts