Observational Study of Velmanase Alfa in Young Children with Alpha-Mannosidosis
This observational study aims to understand how the drug velmanase alfa (Lamzede®) works in the bodies of children under 3 years old who have Alpha-Mannosidosis, a rare genetic disorder. Researchers want to see how the drug affects a specific marker of the disease called GlcNAc(Man)2 after one year of treatment. They also want to learn how your child's body reacts to velmanase alfa during therapy. Your child can join if they have a confirmed diagnosis of Alpha-Mannosidosis, started velmanase alfa treatment before age 3, and have information on the GlcNAc(Man)2 marker from before treatment. The study plans to include 5 participants.
- Study design
- This is an observational study, meaning researchers will gather information from routine care rather than conducting new tests. It aims to enroll 5 participants.
- What's involved
- Your legal guardian will be asked to provide results from routine clinical tests related to your general health and velmanase alfa administration.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary goal is to measure the drug's effect after 52 weeks (one year) of treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
At a glance
Conditions
Where it's being run
2 sites across 2 statesWho to contact
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Do you actually qualify for this trial?
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Inclusion
What this trial measures
- Pharmacodynamic Response to velmanase alfa52 weeks of treatment
Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood