Project
A multinational clinical project (OligoG-pivotal-CF) to test the orphan drug OligoG CF-5/20 in a pivotal phase 2b clinical trial for the treatment of cystic fibrosis.
Details
The clinical trial will include 120 CF patients from 45 European sites in coordination with the European Clinical Trial Network. It is anticipated that the project will deliver a new and improved therapeutic approach within cystic fibrosis that will be available for CF patients by 2021.
Funded by the European Commission H2020 programme (Personalized Medicine).
Project start: January 2018.
Patient enrollment begins: H1 – 2019
Project duration: 36 months
The project aims to bring AlgiPharmas’s drug candidate OligoG to market as a first in class calcium chelating mucus releasing drug for treatment of CF. Based on the available phase 1 and 2 clinical data, a pivotal dose finding phase 2b clinical trial assessing long term efficacy and safety of OligoG in CF is at the core of the project.
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.
Challenge
Cystic fibrosis is a life-threatening disease that affects the lungs and digestive system and impacts about 70,000 people worldwide.
Status
The project status will be updated periodically to reflect the progress of the clinical study. Patient enrolment is due to start H1 – 2019.
Partners
Resources
The project status will be updated periodically to reflect the progress of the clinical study. Patient enrolment is due to start H1 – 2019. On our resources page we will post related articles and link to other websites with information about CF that would be useful to patients, doctors and scientists.
Cystic Fibrosis
National patient support, advocacy and research resources
Clinical trials
Information related to clinical trials
CFTR
Other background on CFTR
Literature
Scientific and clinical literature
EUROPEAN CLINICAL TRIAL NETWORK
The clinical trial will include 120 CF patients from 45 European sites in coordination with the European Clinical Trial Network.
45
European sites
120
CF patients
Funding
This project has received funding from the European Union’s Horizon 2020 research and innovation programme part “Health, demographic change and well-being” (call H2020-SC1-2016-2017) under grant agreement No 755234.