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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:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacodynamic Response to velmanase alfa | Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood | 52 weeks of treatment |
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| Measure | Description | Time Frame |
|---|---|---|
| Development of anti drug antibody to velmanase alfa | Assessment of the impact of anti drug antibody on pharmacodinamic | 52 weeks of treatment |
| Treatment-emergent adverse events | 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 |
Inclusion Criteria:
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).
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Paediatric patients with a confirmed diagnosis of alpha mannosidosis via MAN2B1 genetic testing and/or alpha-mannosidase activity in leukocytes or fibroblasts < 10% of normal activity, with data for at least one pre- and one post-Lamzede treatment sample obtained when < 3 YOA, after at least 6 weeks of treatment (primary analyses).
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Chiesi Clinical trials | Contact | +39.0521 2791 | clinicaltrials_info@chiesi.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nicklaus Children's Hospital | Recruiting | Miami | Florida | 33155 | United States |
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| Label | URL |
|---|---|
| Chiesi site web study information | View source |
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| ID | Term |
|---|---|
| D008363 | alpha-Mannosidosis |
| ID | Term |
|---|---|
| D044904 | Mannosidase Deficiency Diseases |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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|
| 52 weeks |
| Greenwood Genetic Center | Recruiting | Greenwood | South Carolina | 29605 | United States |
|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |