Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
NCT ID: NCT06184503
Last Updated: 2025-05-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
5 participants
OBSERVATIONAL
2025-02-19
2029-09-30
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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OTHER
OTHER
Study Groups
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Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age
Paediatric patients with a confirmed diagnosis of alpha-mannosidosis with data for at least one pre- and one post-Lamzede treatment sample obtained when \< 3 YOA.
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.
Interventions
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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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
3 Years
ALL
No
Sponsors
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Chiesi Farmaceutici S.p.A.
INDUSTRY
Responsible Party
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Locations
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Nicklaus Children's Hospital
Miami, Florida, United States
Greenwood Genetic Center
Greenwood, South Carolina, United States
Countries
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Central Contacts
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Facility Contacts
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Related Links
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Chiesi site web study information
Other Identifiers
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CLI-LMZYMAA2-04
Identifier Type: -
Identifier Source: org_study_id
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