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NCT02998879: rhLaman-08
A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-Mannosidosis
Phase 2 trial testing Velmanase Alfa (e.g. Lamazym) in Alpha-Mannosidosis in 5 participants. Completed in 1 July 2020.
1 July 2020
Quick facts
| Lead sponsor | Chiesi Farmaceutici S.p.A. |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 5 |
| Start date | 1 December 2016 |
| Primary completion | 1 July 2020 |
| Estimated completion | 1 July 2020 |
| Sites | 6 locations across Austria, Denmark, France, Germany, Italy |
Drugs / interventions tested
- Velmanase Alfa (e.g. Lamazym) — 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 6, 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.
-
Safety and tolerability of velmanase alfa as per Adverse events
Time frame: From baseline throughout study completion, at least of 2 years
Safety and tolerability assessed as per AEs including infusion-related reactions \[IRRs\] -
Safety and tolerability of velmanase alfa as per vital signs
Time frame: From baseline throughout study completion, at least of 2 years -
Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per hematology
Time frame: From baseline throughout study completion, at least of 2 years -
Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per blood biochemistry
Time frame: From baseline throughout study completion, at least of 2 years -
Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per urinalysis
Time frame: From baseline throughout study completion, at least of 2 years -
Detection of anti-velmanase alfa-IgG antibodies (ADA) and neutralizing/inhibitory antibodies
Time frame: From baseline throughout study completion, at least of 2 years
Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained
Sponsor's own description
The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
-
Early biochemical effects of velmanase alfa in a 7-month-old infant with alpha-mannosidosis.
Santoro L, Zampini L, Padella L, Monachesi C, et al · · 2020 · cited 9× · PMID 32905047 · DOI 10.1002/jmd2.12144 -
Extended long-term efficacy and safety of velmanase alfa treatment up to 12 years in patients with alpha-mannosidosis.
Guffon N, Borgwardt L, Tylki-Szymańska A, Ballabeni A, et al · · 2025 · cited 6× · PMID 39381850 · DOI 10.1002/jimd.12799 -
Can velmanase alfa be the next widespread potential therapy for alpha-mannosidosis?
Abdul Ghani S, Burney S, Ul Hussain H, Abdul Wahid M, et al · · 2023 · cited 2× · PMID 37352513 · DOI 10.1097/js9.0000000000000528
Verify or expand the search:
- PubMed search for NCT02998879
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Alpha-Mannosidosis
Currently open trials in the same condition.
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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 NCT02998879 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- 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: 18 October 2021
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/NCT02998879.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing