Australian specialist pharmaceutical company Pharmaxis has just announced it has successfully enrolled the first participant in an international Phase 3 clinical trial designed to evaluate their lead pipeline product for cystic fibrosis, Bronchitol® (mannitol). Pharmaxis continues to develop novel treatments for chronic respiratory disorders, such as asthma, pulmonary fibrosis, lung cancer, and COPD.
Cystic fibrosis (CF) is a genetic disease that commonly affects the respiratory and digestive tract by causing the production of excessive, thick mucus. Inhaled mannitol, an osmotic agent, can help draw water into lung surfaces to help water down and thin out stubborn mucus build up, in order to facilitate physiotherapy in CF patients.
This ongoing clinical trial was designed to meet the standards set forth by the US Food and Drug Administration (FDA) for the potential approval of Bronchitol in the US. Under FDA stipulations, the Phase 3 study will be carried out quite similarly to a pair of previous Pharmaxis clinical trials, namely CF301 and CF 302, and will run for 26 weeks. It is classified as a randomized, placebo-controlled, double blind parallel group research of the drug and will be given as a 400mg dose, twice a day in adult CF patients. The company’s researchers are aiming to enroll 350-440 participants aged 18 years and older, who will be assessed for improvement based on the following parameters: pulmonary function, incidences of exacerbations, and safety.
INC, an international contract research organization, will be carrying out Pharmaxis remote investigations. There are over 100 study sites in several locations across 19 countries: 12 in Europe, 2 each in North and South America, 1 in South Africa, and Australia and New Zealand. The company expects it will take about a year for the clinical trial to be fully enrolled.
“This Phase 3 trial in adults has been carefully designed with the benefit of our two previous CF trials (CF301 and CF 302) where a post hoc analysis1 of the subgroups of adult patients showed a significant improvement in FEV1,” Gary Phillips, Chief Executive Officer, Pharmaxis. “The design of the trial also incorporates the very clear guidance provided by the FDA concerning what is required in order to gain approval for Bronchitol in the US. We are very pleased to have enrolled the first patient.”
The drug is a spray-dried formulation, inhaled via a portable inhaler, specifically designed for this form of osmotherapy. Today, Bronchitol is approved in Australia for use in CF patients older than 6 years old, and in the European Union and Israel for adult patients.
Bronchitol is a precision spray-dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler. The product is approved for marketing for patients aged over six years in Australia and for patients aged 18 years and over throughout the European Union and in Israel.
Those interested in learning how one can participate in this study can contact Felicity Moffatt through +61 418 677 701 or email [email protected].