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

Chiesi

Status: Enrolling

Conditions: Alpha-Mannosidosis

Treatments: Drug: Velmanase Alfa

Study type: Observational

Funder types: Industry

Identifiers

NCT06184503

CLI-LMZYMAA2-04

Details and patient eligibility

About

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.

Enrollment: 5 estimated patients

Sex: All

Ages: Under 3 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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.

Exclusion criteria

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

Trial design

5 participants in 1 patient group

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

Description:

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.

Treatment:

Drug: Velmanase Alfa