ClinicalTrials.gov record NCT06184503

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 25, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
AgeUp to 3 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational study collects routine clinical and lab data to learn how velmanase alfa (Lamzede®) affects a disease marker in young children. It focuses on children who began treatment at least six weeks before their third birthday and compares marker levels from before treatment and after, with a primary measurement at about 52 weeks. Data may also be taken from registries, compassionate-use programs, other studies, and published case reports.

What it is studying. The study tests how blood levels of the marker GlcNAc(Man)2 change after starting velmanase alfa, with the main outcome measured at about 52 weeks of treatment. It also explores pharmacodynamic responses — how the child's body reacts to the drug — using results collected in routine clinical care and from other data sources.

What the researchers hope to learn. To measure change in the blood marker GlcNAc(Man)2 after velmanase alfa treatment and to describe pharmacodynamic responses in children treated before age 3.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Velmanase Alfa

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Parents or legal guardians will provide results from routine clinical care related to the child's health and velmanase alfa treatment, including measurements of the GlcNAc(Man)2 disease marker before and after treatment. The study uses existing clinical data and may also extract relevant information from registries, compassionate use programs, investigator-initiated studies, or published case reports. The main analysis looks at change in GlcNAc(Man)2 after about 52 weeks of treatment; no additional invasive procedures beyond those done as part of routine care are specified in the record.

How the study is structured. An observational study using existing clinical records and lab results; no treatments are assigned by the study. It uses each participant's pre-treatment and post-treatment biomarker measurements (including a primary comparison around 52 weeks) and may include data drawn from other registries, compassionate-use programs, investigator‑initiated studies, or published case reports.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on alpha-mannosidosis

Clinical trials6 trials listed for alpha-mannosidosisEvery trial the registry lists for alpha-mannosidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in alpha-mannosidosis researchLatest monthly update: September 2026. Includes: Everyday treatment effects. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Feb 19, 2025
Primary completion
September 2029 (estimated)
Study completion
September 2029 (estimated)
Target enrollment
5 participants (estimated)
Last updated on registry
May 16, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Chiesi Farmaceutici S.p.A.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (2)

Terms used on this page · explanations by MedicalUpdates.ai

compassionate use
A way for some people with serious conditions to receive an investigational treatment outside a clinical trial, under specific rules. It is not itself a clinical trial and is not designed to test whether the treatment works.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
LAR
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
HSCT
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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