LAMPO (0‑3): Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha‑Mannosidosis Under the Age 3
About the Condition
Alpha-mannosidosis is a very rare condition known as a lysosomal storage disorder. It is a chronic disease inherited through genes passed down from parents to their children. Alpha-mannosidosis affects about 1 in 500,000 to 1 in 1,000,000 live births.
Alpha Mannosidosis can cause children and adults to suffer from skeletal deformities, coarse facial features, hearing loss, cognitive disabilities, problems with the immune system (which may mean, for example, you catch infections more readily), mental health and behavioural issues. Alpha-mannosidosis is what’s considered a “progressive disease,” meaning it changes or gets worse over time.
Trial Details
LAMPO (0-3) is an observational study. 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. In observational studies, researchers watch participants as they receive their usual treatments or go about their everyday lives. They track health outcomes over time without changing participants’ routines or adding new treatments. While these studies don't test new treatments directly, they help uncover insights and patterns that can identify potential treatments or prevention strategies.
The main questions the researchers aim to answer are:
- what are the effects of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after up to one year of therapy ?
- how the child's body reacts to velmanase alfa during the therapy?
Trial Participation
In this study, the parents or legal guardians of children treated with velmanase alfa will be asked to provide the results of analyses performed in the routine clinical setting related to the child's general health and the administration of velmanase alfa.
Additional information will be extracted by researchers from other studies/registries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature).
Trial Participant Requirements
Key Inclusion and Exclusion Criteria
Participants Must:
- 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 Must Not:
- Have received prior blood stem cell transplant or other investigational therapies for the treatment of alpha mannosidosis (supportive treatments acceptable).
- 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.
- Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).