Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3 (LAMPO(0-3))

A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Alpha-Mannosidosis
• Intervention / Treatment: Drug: Velmanase Alfa

• Study Type: Observational
• Enrollment (Estimated): 5

Contacts and Locations

• Study Contact: Name: Chiesi Clinical trials; Phone Number: +39.0521 2791; Email: clinicaltrials_info@chiesi.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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).

Description

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).

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	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.; Other Names: Lamzede

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Other Outcome Measures

Outcome Measure	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	52 weeks

Collaborators and Investigators

• Sponsor: Chiesi Farmaceutici S.p.A.

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2024
• Primary Completion (Estimated): September 1, 2029
• Study Completion (Estimated): September 1, 2029

Study Registration Dates

• First Submitted: December 14, 2023
• First Submitted That Met QC Criteria: December 14, 2023
• First Posted (Actual): December 28, 2023

Study Record Updates

• Last Update Posted (Actual): December 28, 2023
• Last Update Submitted That Met QC Criteria: December 14, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mannosidase Deficiency Diseases; alpha-Mannosidosis
• Other Study ID Numbers: CLI-LMZYMAA2-04

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No