An influential group of European cystic fibrosis experts has signed off on the design of a Phase 3 clinical trial of Sollpura (lipromatase) as a treatment for a CF-related digestive disorder known as exocrine pancreatic insufficiency.
The European Cystic Fibrosis Society Clinical Trial Network’s approval will speed up the recruitment of participants, according to Sollpura’s developer, Anthera Pharmaceuticals. The sign-off came from the organization’s executive committee.
A similar U.S. organization, the Cystic Fibrosis Foundation Therapeutics Development Network, has also approved the design of the RESULT trial (NCT03051490).
The sooner Anthera reaches its goal of recruiting 150 participants, the sooner it can start the trial and make the results available. It hopes to release the findings at the end of 2017 or the beginning of 2018.
CF experts estimate that 90 percent of patients have exocrine pancreatic insufficiency, or EPI. The disorder makes it difficult for a person to digest food.
A hallmark of CF is overly thick mucus in a number of places in the body. The pancreas produces juices that help the small intestine digest food, but thick mucus in the channels that deliver the juices to the intestine can prevent them from getting there. This means CF patients can have difficulty obtaining enough nutrients.
Those conducting the trial will tailor doses of Sollpura to individual patients’ needs, to try to achieve the best results.
One of the things the trial will evaluate is whether Sollpura is as good as Pancreaze, an approved treatment for the condition that is made from purified pig proteins.
Both drugs are pancreatic enzyme replacement therapies, meaning they replace enzymes the body normally produces in the pancreas.
Because Sollpura is synthetic, it is easier to produce in large amounts than Pancreze, preventing supply problems. A synthetic product also prevents the risk of contamination from a pig-associated virus or other infectious agent.
In addition, Pancreaze must be taken in capsule form to protect it as it goes through the stomach. Sollpura does not. Anthera is developing a drinkable formulation that would make it easier to give the drug to infants and children, or to patients who use feeding tubes.
“We are very pleased” that the European trial network has approved the design of the study, Craig Thompson, president and CEO of Anthera, said in a press release. “This achievement, in combination with the earlier approval from the Cystic Fibrosis Foundation Therapeutics Development Network, represents another key milestone for the RESULT trial.”