Packages

Work Package 6

Clinical trial Phase III

Involves Partners ZYMX, CMMC, UMG, HCL, DTI, Unilabs and Larix

Objectives:
The phase III trial will be performed to gather additional information about efficacy and safety and to evaluate the overall benefit/risk profile of rhLAMAN as a therapeutic drug for treatment of alpha-Mannosidosis

Description of the Work:
Once the effective dose has been established within phase II, a randomized and placebo controlled Phase III trial will be initiated at four clinical sites in France, Poland, United Kingdom and Germany.
Procedure:
A minimum of 20 patients will be enrolled in this 6 months trial and as this phase III trial will be placebo-controlled, 20% of the patients will be treated with the placebo. In this trial the aim is primarily to recruit patients which are residents in one of the four countries and already are patients at the site. This will reduce the travel costs and the complexity of the trial. Clinical Operations at Zymenex will then have experience with all the assessments and can provide a powerful support to the sites. The doses are based on the minimum effctive dose established in phase IIa.

Dosing will be weekly over a period of 6 months. Efficacy on functional capacity in the trials will be assessed by

change in endurance (6-minute walk test),
improved lung function (absolute FVC) and
reduction of oligosaccharides in urine and serum.