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

Part of paid clinical trials in Miami, Florida.

Sponsor
Chiesi Farmaceutici S.p.A.
Study ID
NCT06184503
Status
Recruiting
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Conditions

  • Alpha-Mannosidosis

Eligibility Criteria

Sex
ALL
Age
N/A - 3 Years
Healthy Volunteers
Not accepted

Interventions

  • Velmanase Alfa — DRUG
    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.

Study Details

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.

Key Dates

Start date
Feb 19, 2025
Status verified
May 2025
Primary completion
Sep 30, 2029
Completion
Sep 30, 2029

Study Design

Enrollment
5 participants (estimated)

Arms

  • Arm: 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.

Primary Outcome Measure

Pharmacodynamic Response to velmanase alfa [ Time Frame: 52 weeks of treatment ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Nicklaus Children's HospitalMiamiFlorida33155
Parul Jayakar
[email protected]
786-624-4717
Greenwood Genetic CenterGreenwoodSouth Carolina29605
Laura Buch
[email protected]
864-250-7944

Find similar trials in Miami, FL

By research site
Nicklaus Children's Hospital· Miami, FLGreenwood Genetic Center· Greenwood, SC

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