Effects of Velmanase Alfa in Young Children with Alpha-Mannosidosis
Official title: A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age
Run by Chiesi Farmaceutici S.p.A. · NCT06184503 · Data updated 2026-11-12
This study is testing how well the drug velmanase alfa works for young children under 3 with Alpha-Mannosidosis to see if it helps improve their health after a year of treatment.
Quick facts
| Study type | Observational |
|---|---|
| Ages | Any age to 3 Years |
| Sex | All |
| Enrollment | 5 (estimated) |
| Sponsor | Chiesi Farmaceutici S.p.A. Industry-sponsored |
| Locations | 2 sites (Miami, Florida and 1 other locations) |
| Status | Recruiting |
| Started | February 19, 2025 |
| Main results expected | September 2029 |
| Study ends | September 2029 |
| Registry record updated | May 16, 2025 |
| Trial ID | NCT06184503 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are children under 3 years old with a confirmed diagnosis of Alpha-Mannosidosis who have started treatment with velmanase alfa.
✕Probably not a fit
Patients who have previously undergone hematopoietic stem cell transplantation or other investigational therapies for Alpha-Mannosidosis may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study aims to assess the effects of velmanase alfa (Lamzede®) on children under the age of 3 diagnosed with Alpha-Mannosidosis. It will evaluate the drug's impact on a specific disease marker, GlcNAc(Man)2, after one year of therapy and monitor how the children's bodies respond to the treatment. Data will be collected from routine clinical analyses and other observational studies to provide a comprehensive understanding of the drug's effects.
Why it matters
Potential benefit: If successful, this study could provide insights into the effectiveness of velmanase alfa in managing Alpha-Mannosidosis in young children.
How similar studies have gone: While this approach is observational, similar studies have shown promise in understanding treatment effects in rare diseases, but the specific use of velmanase alfa in this age group is novel.
Who can join: the full rules
Show full inclusion / exclusion 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).
Where it's running
-
Nicklaus Children's Hospital
Miami, Florida, United States Recruiting -
Greenwood Genetic Center
Greenwood, South Carolina, United States Recruiting
Study contacts
- Study coordinator: Chiesi Clinical trials
- Email: clinicaltrials_info@chiesi.com
- Phone: +39.0521 2791
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Long-term observation of rare genetic brain disorders
This study is tracking how rare genetic brain disorders progress over time to better understand their effects on patients.
-
Using umbilical cord blood cells to treat inherited metabolic diseases
This study is testing if a new treatment using special cells from umbilical cord blood can help people with inherited metabolic diseases and early signs of nerve damage feel better when combined with standard cord blood transplants.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.