NCT06184503 Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
| NCT ID | NCT06184503 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Chiesi Farmaceutici S.p.A. |
| Condition | Alpha-Mannosidosis |
| Study Type | OBSERVATIONAL |
| Enrollment | 5 participants |
| Start Date | 2025-02-19 |
| Primary Completion | 2029-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 5 participants in total. It began in 2025-02-19 with a primary completion date of 2029-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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).
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06184503 clinical trial?
This trial is open to participants of all sexes, up to 3 Years, studying Alpha-Mannosidosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06184503 currently recruiting?
Yes, NCT06184503 is actively recruiting participants. Contact the research team at clinicaltrials_info@chiesi.com for enrollment information.
Where is the NCT06184503 trial being conducted?
This trial is being conducted at Miami, United States, Greenwood, United States.
Who is sponsoring the NCT06184503 clinical trial?
NCT06184503 is sponsored by Chiesi Farmaceutici S.p.A.. The trial plans to enroll 5 participants.