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Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3

NCT06184503 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 5

Last updated 2025-05-16

No results posted yet for this study

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 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 The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa.

Additional data will be extracted from other observational sponsored studies/registries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing.

Conditions

  • Alpha-Mannosidosis

Interventions

DRUG

Velmanase Alfa

Lamzede® (velmanase alfa, henceforth referred to as Lamzede) is a recombinant human lysosomal alpha-mannosidase product developed as an intravenous enzyme replacement therapy (ERT) for the treatment of alpha-mannosidosis.

Sponsors & Collaborators

  • Chiesi Farmaceutici S.p.A.

    lead INDUSTRY

Eligibility

Max Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-02-19
Primary Completion
2029-09-30
Completion
2029-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06184503 on ClinicalTrials.gov