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NCT06184503: LAMPO(0-3)
A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age
trial testing Velmanase Alfa in Alpha-Mannosidosis in 5 participants. Currently enrolling.
1 September 2029
Quick facts
| Lead sponsor | Chiesi Farmaceutici S.p.A. |
|---|---|
| Status | Recruiting now |
| Study type | OBSERVATIONAL |
| Enrollment | 5 |
| Start date | 19 February 2025 |
| Primary completion | 1 September 2029 |
| Estimated completion | 1 September 2029 |
| Sites | 2 locations across United States |
Drugs / interventions tested
- Velmanase Alfa — full drug profile →
Conditions studied
- Alpha-Mannosidosis — all drugs for Alpha-Mannosidosis →
Sponsor
Chiesi Farmaceutici S.p.A. — full company profile →
Who can join
Under 3, any sex, with Alpha-Mannosidosis. Patients with the condition only — healthy volunteers not accepted.
What's being measured
Primary outcomes are the specific endpoints the trial is designed to prove or disprove.
-
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
Sponsor's own description
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.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT06184503
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Velmanase Alfa
Trials testing the same drug.
- NCT04031066 — Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis · Phase 3 · withdrawn
- NCT04959240 — Expanded Access to Velmanase Alfa · available
Other recruiting trials for Alpha-Mannosidosis
Currently open trials in the same condition.
- NCT02171104 — MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis · Phase 2 · active not recruiting
Other Chiesi Farmaceutici S.p.A. trials
Trials by the same sponsor.
- NCT07516951 — A Study to Find an Efficacious and Safe Dose of CHF10067 (Zampilimab) in Participants With Idiopathic Pulmonary Fibrosis · Phase 2 · not yet recruiting
- NCT07301736 — Therapeutic Equivalence of CHF5993 pMDI 100/6/12.5 µg HFA-152a in Subjects With Mild to Moderate Asthma · Phase 2 · recruiting
- NCT07163182 — Evaluation of Safety, Side Effects and How the Drug CHF6467 Administered Via Intranasal Route is Absorbed, Modified and · Phase 1 · recruiting
- NCT06900816 — A Comparison Study Between Adolescents With Asthma and Adults With Asthma on How They Absorb, Metabolise and Eliminate C · Phase 2 · completed
- NCT06892756 — Effect of Cyclosporine Drug Interaction on the Absorption, Metabolism and Elimination of CHF6001 in Healthy Volunteers. · Phase 1 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06184503 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Chiesi Farmaceutici S.p.A.
- Last refreshed: 15 May 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06184503.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing