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

Alpha-Mannosidosis Clinical Trial

— LAMPO(0-3)  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06184503
• Other study ID #: CLI-LMZYMAA2-04
• Status: Not yet recruiting
• Start date: May 2024
• Est. completion date: September 2029

Study information

• Verified date: December 2023
• Source: Chiesi Farmaceutici S.p.A.
• Contact: Chiesi Clinical trials
• Phone: +39.0521 2791
• Email: clinicaltrials_info[@]chiesi.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 5
• Est. completion date: September 2029
• Est. primary completion date: September 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 3 Years

Eligibility

Inclusion Criteria:

• Participants with the provision of informed consent from their legal guardians
• Confirmed diagnosis of alpha mannosidosis
• Velmanase alfa treatment initiation from birth to at least six weeks before turning 3

YOA, where the standard of care follow-up includes the following:

• Data for GlcNAc(Man)2 levels obtained when the participant was < 3 YOA for:
• At least one pre-treatment sample obtained no more than 13 weeks before initiating velmanase alfa treatment, 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).

Related Conditions & MeSH terms

• Alpha-Mannosidosis

Intervention

Drug:
• Velmanase 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.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Chiesi Farmaceutici S.p.A.

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Development of anti drug antibody to velmanase alfa	Assessment of the impact of anti drug antibody on pharmacodinamic	52 weeks of treatment
Other	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	52 weeks
Primary	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