Date: 2012-12-12
Type of information: Initiation of preclinical development
phase: 3
Announcement: initiation of the study
Company: Zymenex (Denmark)
Product: rhLAMAN (Lamazym™)
Action mechanism:
Disease: alpha-mannosidosis
Therapeutic area: Rare diseases - Genetic diseases
Country: Denmark, Germany, France, Spain, Belgium, Norway
Trial details:
Latest
news: * On December 12, 2012, Zymenex has announced that the biotechnologically derived human enzyme product rhLAMAN (Lamazym™) has been successfully evaluated in a 12 month dose/response Phase 2a clinical trial and a 6 month follow-up Phase 2b clinical trial in 9 patients. The aim of the trials, which took place at the Department of Clinical Genetics, Copenhagen University Hospital, was to confirm the chosen weekly minimum effective dose of 1 mg/kg and to validate the clinical effect of repeated i.v dosing. Positive changes from baseline after 18 months of dosing have been demonstrated in the primary measured clinical and biochemical endpoints.
Statistically significant changes were demonstrated in oligosaccharide levels in serum, in the functional tests: 3 Minute Stair Climb (+39 steps, p=0.004) and 6 Minute Walk Test (+71 meters, p=0.009) as well as in the Leiter-R test of intellectual capacity (+0.5, p=0.022). The forced vital capacity (FVC), lung function test showed a marked increase (+9.2%, p=0.157) .
A 12-month, Multi-Center, Double-Blind, Randomized and Placebo-Controlled trial in 25 patients with alpha-Mannosidosis, receiving a dose of 1 mg/kg weekly, is now been initiated in Denmark, Germany, France, Spain, Belgium and Norway. The dosing will take place in the patient’s home countries and all evaluations will be
performed at the Department of Clinical Genetics, Copenhagen University Hospital, in Denmark.
* On January 20, 2012, Zymenex has announced that rhLAMAN (Lamazym™) has successfully completed the dose/response Phase 2a clinical trial and has now initiated a 6 month Phase 2b clinical trial in 9 patients, where the aim is to confirm the chosen minimum effective dose of 1 mg/kg dosed weekly and to validate the clinical
effect of repeated i.v dosing. Phase 2a trial demonstrated that the enzyme is clinically effective, safe and well
tolerated and a confirmatory Phase 2b clinical trial is now underway.
* On October 11, 2011, Zymenex has announced that rhLAMAN (LamazymTM) has successfully completed a Phase 2a clinical trial. “9 patients aged 7 to 18 years were recruited to the Phase 2a clinical trial from European hospitals and each week flown to Copenhagen, Denmark to be treated here at the Department of Clinical Genetics, Copenhagen University Hospital”, says Chief Physician Dr. Allan Meldgaard Lund MD, who is the Principal Investigator and treating physician.
The patients were divided into two cohorts receiving doses of 1 mg/kg or 2mg/kg respectively each week. Apart from finding the minimum effective dose, the goal of the Phase 2a trial was to demonstrate that the enzyme was effective and improved the patient’s condition. There was a clinically relevant improvement in the parameters
measured and thus the goal has been achieved.
This now allows the 9 patients to be moved forward into a 6 -month Phase 2b clinical trial, where the aim is to verify the minimum effective chosen dose of 1mg/kg, by monitoring the effect on chosen composite efficacy endpoints in order to confirm the clinical effect of repeated weekly i.v dosing.
* On January 25, 2011, Zymenex has announced that rhLAMAN (Lamazym™) has successfully completed Phase 1 trials and has now entered Phase 2a clinical trials. The patients were recruited into the Phase 1 trial from around Europe, where the initial goal of demonstrating that the enzyme is safe and well tolerated, has been achieved. This now allows the 10 patients to be moved forward into a 6-month Phase 2a dose-finding clinical trial, where the aim is to identify the most optimal dose to achieve the desired clinical effect.
