The overall objective of the project is to deliver OligoG as a new orphan drug attacking the primary phenotypic effect of the genetic disease cystic fibrosis to the European market, through a dose finding, pivotal phase 2b clinical trial. The trial will be designed to enable preparation of a conditional market authorisation application (cMAA) for Europe, for the treatment of cystic fibrosis lung disease. The study objectives are:
Primary objective: Identify the dose of OligoG for optimal efficacy and safety. The decision will be made on the basis of safety parameters as well as results for the primary endpoint FEV1, supported by secondary endpoints including other spirometry data, microbiology and rheology.
Secondary objectives: Assess the long term safety of OligoG as compared to placebo in patients with cystic fibrosis, and characterize the exposure-response relationships for measures of effectiveness and toxicity in a subgroup of patients.