Recruiting

Velmanase Alfa

Sponsor:

Chiesi Farmaceutici S.p.A.

Code:

NCT06184503

Conditions

Alpha-Mannosidosis

Eligibility Criteria

Sex: All

Age: 0 - 3

Healthy Volunteers: Not accepted

Interventions

Velmanase Alfa

Study Details

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

Study ID

NCT06184503

Start date

Feb 19, 2025

Status verified date

May, 2025

Completion date

Sep, 2029

Anticipated

Primary completion date

Sep, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 3

Healthy Volunteers: Not accepted

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).

Study Design

Enrollment

5 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Interventions

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.

Primary outcome measure

  • Pharmacodynamic Response to velmanase alfa [ Time Frame: 52 weeks of treatment ]

Central Contacts and Locations

Central contacts

Chiesi Clinical trials

+39.0521 2791clinicaltrials_info@chiesi.com

Locations

Nicklaus Children's Hospital

Recruiting

Miami, Florida, United States, 33155

Contacts

Parul Jayakar

786-624-4717Parul.Jayakar@Nicklaushealth.org

Greenwood Genetic Center

Recruiting

Greenwood, South Carolina, United States, 29605

Contacts

Laura Buch

864-250-7944lbuch@ggc.org

More Information

Sponsor

Chiesi Farmaceutici S.p.A.

Last update posted

May 16, 2025

Last verified

May, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Chiesi Farmaceutici S.p.A. on 2025-05-16.