Alpha-Mannosidosis
Conditions
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.
Interventions
DRUGVelmanase 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.
Sponsors
Chiesi Farmaceutici S.p.A.
Study design
Observational model
OTHER
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
No minimum to 3 Years
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Development of anti drug antibody to velmanase alfa | 52 weeks of treatment | Assessment of the impact of anti drug antibody on pharmacodinamic |
| Treatment-emergent adverse events | 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 |
Countries
United States
Contacts
Primary ContactChiesi Clinical trials
Outcome results
None listed