NCT01268358

A Single Center, Open-label, Dose Escalation Study of the Safety and Pharmacokinetics of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.

Completed Phase 1 Last updated 30 July 2020

What this trial tests

Phase 1 trial testing Lamazym in Alpha Mannosidosis in 10 participants. Completed in 1 January 2011.

Timeline

1 October 2010

Primary endpoint
1 January 2011

1 January 2011

Quick facts

Lead sponsor	Zymenex A/S
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	1 October 2010
Primary completion	1 January 2011
Estimated completion	1 January 2011

Drugs / interventions tested

• Lamazym — full drug profile →

Conditions studied

• Alpha Mannosidosis — all drugs for Alpha Mannosidosis →

Sponsor

Zymenex A/S — full company profile →

Who can join

Adults 5 to 20, 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.

• To evaluate the safety profile of rhLAMAN (Lamazym)
  Time frame: 1-5 weeks

Sponsor's own description

This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym. In order to avoid development of delayed hypersensitivity all patients will continue weekly treatment at the designated dose until the Safety Committee approves transfer to the rhLAMAN-03 protocol.

It is the hypothesis that Lamazym is safe to use.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial.
   Borgwardt L, Guffon N, Amraoui Y, Dali CI, et al · · 2018 · cited 43× · PMID 29846843 · DOI 10.1007/s10545-018-0185-0
2. 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
3. Relationship between <i>MAN2B1</i> genotype/subcellular localization subgroups, antidrug antibody detection, and long-term velmanase alfa treatment outcomes in patients with alpha-mannosidosis.
   Borgwardt LG, Ceravolo F, Zardi G, Ballabeni A, et al · · 2023 · cited 5× · PMID 36873087 · DOI 10.1002/jmd2.12349
4. 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