Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06184503

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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Alpha-Mannosidosis Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lysosomal Storage Diseases Mannosidase Deficiency Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

Up to 3 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Nicklaus Children's Hospital, Miami, Florida, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

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.

Other names: Lamzede

Primary outcomes

  1. Pharmacodynamic Response to velmanase alfa

    Time frame: 52 weeks of treatment

    Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood

Other outcomes

  1. Development of anti drug antibody to velmanase alfa

    Time frame: 52 weeks of treatment

    Assessment of the impact of anti drug antibody on pharmacodinamic

  2. Treatment-emergent adverse events

    Time frame: 52 weeks

    Number of undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the treatment

Study contacts

Contact information is provided by the study sponsor or research team.

Chiesi Clinical trials

CONTACT

[email protected]

+39.0521 2791

Sponsors and collaborators

Lead sponsor

Chiesi Farmaceutici S.p.A.

Industry

Registry information

Official study title

A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age

Acronym: LAMPO(0-3)

Important dates

Study start
2025
Primary completion
2029
Study completion
2029
First posted
Dec 28, 2023
Registry last updated
May 16, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.