NCT02478840: rhLAMAN-10

A Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) Treatment in Subjects With Alpha-Mannosidosis Who Previously Participated in Lamazym Trials

Completed Phase 3 Last updated 18 November 2020

What this trial tests

Phase 3 trial testing Lamazym in Alpha-Mannosidosis in 18 participants. Completed in 1 June 2015.

Timeline

1 February 2015

Primary endpoint
1 June 2015

1 June 2015

Quick facts

Lead sponsor	Zymenex A/S
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	18
Start date	1 February 2015
Primary completion	1 June 2015
Estimated completion	1 June 2015
Sites	1 location across Denmark

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

Eligibility, 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.

• Change from baseline in reduction of oligosaccharides in serum
  Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
  Primary Endpoint evaluation as change
• Change from baseline in 3 Minutes Stair Climb Test (3MSCT)
  Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
  Primary Endpoint evaluation as change

Sponsor's own description

The overall objective is to evaluate the long-term efficacy of Lamazym i.v. treatment in patients with alpha-Mannosidosis previously enrolled in Lamazym trials and currently receiving the treatment according to the AfterCare Program.

Publications & conference data

5 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. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
   Lund AM, Borgwardt L, Cattaneo F, Ardigò D, et al · · 2018 · cited 35× · PMID 29725868 · DOI 10.1007/s10545-018-0175-2
3. 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
4. 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
5. Velmanase alfa approved for treatment of non-central nervous system manifestations of alpha-mannosidosis: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).
   Lander JM, Penon-Portmann M, Sutton VR, Chang I, et al · · 2024 · PMID 39669629 · DOI 10.1016/j.gimo.2024.101832