Ignite Creation Date:
2024-05-06 @ 7:55 PM
Last Modification Date:
2024-10-26 @ 3:16 PM
Study NCT ID:
NCT06184503
Status:
RECRUITING
Last Update Posted:
2023-12-28
First Post:
2023-12-14
Brief Title:
Analysis of Velmanase Alfa Lamzedes Effects in the Body of Children with Alpha-Mannosidosis Under the Age 3
Sponsor:
Chiesi Farmaceutici SpA
Organization:
Chiesi Farmaceutici SpA
Study Overview
Official Title:
A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa Lamzede Treatment in Patients with Alpha-Mannosidosis Less Than 3 Years of Age
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
LAMPO0-3
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 GlcNAcMan2 after one year of therapy
explore how the childs 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 participants general health and the administration of velmanase alfa
Additional data will be extracted from other observational sponsored studiesregistries compassionate use programs investigator-initiated studies IIS and published case reports presented in the literature if existing
The main questions it aims to answer are
study the effect of velmanase alfa on a marker of the disease called GlcNAcMan2 after one year of therapy
explore how the childs 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 participants general health and the administration of velmanase alfa
Additional data will be extracted from other observational sponsored studiesregistries compassionate use programs investigator-initiated studies IIS and published case reports presented in the literature if existing
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None