Alpha-MannosidosisLAMPO(0-3)NCT06184503
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
An observational study of Alpha-Mannosidosis, run by Chiesi Farmaceutici S.p.A. at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages Up to 3 years
- Conditions the study is for
- Alpha-Mannosidosis
- What is being tested?
- Velmanase Alfa
- Type of treatment
- Injection or infusion
- How many people
- 5 participants
- Where
- 2 sitesUnited States
- Study dates
- 19 Feb 2025to Sep 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 3 years
- All sexes
- No healthy volunteers
- Child
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).
Who may be able to join
- 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.
Who cannot join
- Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Greenwood Genetic Center
Greenwood, South Carolina, 29605
Laura Buch, 864-250-7944, lbuch@ggc.org
- Recruiting
Nicklaus Children's Hospital
Miami, Florida, 33155
Parul Jayakar, 786-624-4717, Parul.Jayakar@Nicklaushealth.org
About this study
Official title: A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age
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:
Read more
- 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
- Being tested
- Velmanase Alfa
- Medical subject headings
- alpha-Mannosidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Velmanase AlfaDrug
Also called Lamzede
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Velmanase Alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacodynamic Response to velmanase alfa
Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood
Measured: 52 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2023
- Study start19 Feb 2025
- Primary completionSep 2029 (estimated)
- Study completionSep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha-MannosidosisStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06184503
- Sponsor's protocol ID
- CLI-LMZYMAA2-04
- Acronym
- LAMPO(0-3)
- Status last verified
- May 2025
- First submitted
- 14 Dec 2023
- Last updated
- 16 May 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age
- ClinicalTrials.gov ID
- NCT06184503
- Acronym / sponsor ID
- LAMPO(0-3) / CLI-LMZYMAA2-04
- Conditions
- Alpha-Mannosidosis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Study start
- 19 Feb 2025 (actual)
- Primary completion
- Sep 2029 (estimated)
- Study completion
- Sep 2029 (estimated)
- First posted
- 28 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age | – | Drug: Velmanase AlfaPaediatric 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. |
Who may qualify
- Ages
- Up to 3 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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).
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (1 listed)
- Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Velmanase AlfaAlso: LamzedeLamzede® (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. | Drug | Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age |
Primary outcome
Pharmacodynamic Response to velmanase alfa
Time frame: 52 weeks of treatment
Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2025.
- Chiesi site web study informationLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alpha-Mannosidosis, run by Chiesi Farmaceutici S.p.A. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chiesi Clinical trials (+39.0521 2791, clinicaltrials_info@chiesi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pharmacodynamic Response to velmanase alfa (time frame: 52 weeks of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2025.