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

Recruiting

• Conditions: Alpha-Mannosidosis
• Interventions: Drug: Velmanase Alfa

• Registration Number: NCT06184503
• Lead Sponsor: Chiesi Farmaceutici S.p.A.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-12-14
• Study First Submitted QC: 2023-12-14
• Study First Posted: 2023-12-28
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14
• Study Start: 2024-12
• Primary Completion: 2029-09
• Study Completion: 2029-09

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5

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.

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Exclusion Criteria

Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age	Velmanase Alfa	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 Measures

Name	Time	Method
Pharmacodynamic Response to velmanase alfa	52 weeks of treatment	Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood

Trial Locations

Locations (2)

Greenwood Genetic Center; 🇺🇸 Greenwood, South Carolina, United States

Nicklaus Children's Hospital; 🇺🇸 Miami, Florida, United States