Effects of Velmanase Alfa in Young Children with Alpha-Mannosidosis
A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age
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 |
|---|---|
| Enrollment | 5 (estimated) |
| Ages | N/A to 3 Years |
| Sex | All |
| Sponsor | Chiesi Farmaceutici S.p.A. Industry-sponsored |
| Locations | 2 sites (Miami, Florida and 1 other locations) |
| Trial ID | NCT06184503 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are children under 3 years old with a confirmed diagnosis of Alpha-Mannosidosis who have started treatment with velmanase alfa.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Miami, Florida and 1 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.