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

NCT06184503 | Recruiting FDA Drug

• 1 other identifier: CLI-LMZYMAA2-04
• Study Type: observational
• Target: 5
• Locations: 1 country
• Sites: 2

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.

Conditions

Alpha-Mannosidosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Development of anti drug antibody to velmanase alfa

  52 weeks of treatment

• Treatment-emergent adverse events

  52 weeks

Study Arms (1)

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

Interventions

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.

Also known as: Lamzede

Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age

Eligibility Criteria

• Age: Up to 3 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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).

You may qualify if:

• 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.

You may not qualify if:

• Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).

Sponsors & Collaborators

• Chiesi Farmaceutici S.p.A.: lead

Study Sites (2)

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Greenwood Genetic Center

Greenwood, South Carolina, 29605, United States

RECRUITING

Related Links

• Chiesi site web study information

MeSH Terms

Conditions

alpha-Mannosidosis

Condition Hierarchy (Ancestors)

Mannosidase Deficiency Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Chiesi Clinical trials

CONTACT

+39.0521 2791; clinicaltrials_info@chiesi.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 14, 2023
• First Posted: December 28, 2023
• Study Start: February 19, 2025
• Primary Completion (Estimated): September 1, 2029
• Study Completion (Estimated): September 1, 2029
• Last Updated: May 16, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)