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OligoG-pivotal-CF is a multinational clinical project to test the orphan drug OligoG CF-5/20 (OligoG) in a pivotal phase 2b clinical trial for the treatment of cystic fibrosis (CF). The project is funded by the H2020-SC1-2017-Two-Stage-RTD, topic SC1-PM-08-2017, and represents a new therapy for the rare disease cystic fibrosis (CF). The study drug OligoG CF-5/20 (OligoG) has received an EU Orphan Drug Medicinal Product Designation in 2007 and an FDA Orphan Drug Designation February 2016, both for the treatment of cystic fibrosis
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.
The drug candidate: OligoG is an alginate oligosaccharide extracted, purified and processed from seaweed harvested along the Norwegian coastline. The therapeutic potential of OligoG, a sub-fraction of the alginate, was realized more than 10 years ago. Research and development aimed at developing such oligosaccharides as a therapeutic for cystic fibrosis has been the primary focus of AlgiPharma since the establishment of the company in 2006. The pharmacodynamic profile of the drug product suggests several effects related to the calcium binding properties. This in particular involves release of stagnant mucin to improve mucus clearance, and disruption of biofilm, increasing the microbial susceptibility towards antibiotics.
Study design: The clinical trial at the core of the project has been designed in accordance with EMA guidelines. It will be a multi-center, randomized, double-blind, placebo-controlled, parallel group, dose finding phase 2b study in patients with cystic fibrosis. The trial is divided into two parts, with part 1 designed to identify the best dose, while part 2 is designed to assess the efficacy, safety and tolerability of the inhaled alginate oligosaccharide OligoG compared to placebo after 26 weeks of treatment, followed by a 26 weeks open label extension. Randomised patients will receive daily administrations of OligoG or placebo, both administered in addition to standard of care for 26 weeks. All patients will be offered OligoG in an open label study extension of additional 26 weeks. An Independent Data Monitoring Committee (IDMC) will assess the safety throughout the study, and select the optimal OligoG dose based on efficacy and safety data from an interim analysis performed after all subjects have received 26 weeks of treatment.
|Project amount (€):||6.000.000|