Phase 1 cystic fibrosis trial studying novel enzyme replacement therapy in adults

Learn about Norton Healthcare’s Phase 1 trial for a broad-spectrum enzyme replacement therapy for cystic fibrosis

Patients 18 years of age and older with cystic fibrosis now have an option of participating in a Phase 1 trial of a novel, broad-spectrum, orally delivered enzyme replacement therapy for cystic fibrosis (CF).

The randomized, active-treatment trial is testing the safety, tolerability and effect of four dose levels of ANG003, a compound developed by Anagram Therapeutics, in adult subjects with CF-related exocrine pancreatic insufficiency.

Details of the trial, NCT06052293, are available at

“This new class enzyme replacement therapy is designed to provide unparalleled performance with broad-spectrum composition: lipase for fat, protease for protein, amylase for carbohydrate. If clinical trials are successful, this could be a game-changer for people who require oral enzyme replacement therapy,” said Ronald L. Morton, M.D., a principal investigator in the trial and pediatric pulmonologist with Norton Children’s Pulmonology, affiliated with the UofL School of Medicine.

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The Phase 1 trial is being conducted through the Cystic Fibrosis Therapeutics Development Network at approximately 20 national CF care centers in the U.S. The first patients were enrolled in September.

Cystic fibrosis research at Norton Children’s is made possible by gifts given through the Norton Children’s Hospital Foundation in partnership with Cure CF.

Cystic fibrosis causes breathing issues by clogging lungs with thick mucus. This mucus also clogs the pancreas and hampers the release of digestive enzymes. Over time, this can result in the small intestine becoming unable to digest food completely, meaning the digestive tract has to pass partially undigested food through it. This is a condition called exocrine pancreatic insufficiency (EPI).

Of people with CF, an estimated 90% develop EPI. CF is the second most common cause of EPI, after chronic pancreatitis.

Pancreatic enzyme replacement therapy (PERT) is the current standard of care for people with EPI. PERT helps you break down fat, protein and carbohydrates so your body can absorb nutrients. Since 2009, the Food and Drug Administration has approved six PERT medications, all of them containing pancrelipase, which contains lipase, protease and amylase.

In the Phase 1 trial of ANG003, approximately 48 to 60 eligible subjects will be enrolled in the study, with 12 to 15 subjects assigned to each dose level.

The trial is open to patients 18 and older with a confirmed CF diagnosis, defined as CF signs and symptoms and two CF-causing mutations on genetic testing or sweat chloride greater than 60 milliequivalents per liter. They also must have a body mass index equal to or greater than 20 kilograms per meter squared, a documented history of fecal elastase less than 100 µg/g stool, and EPI clinically controlled, with minimal symptoms and a stable dose of PERT for 90 days before screen.

Anagram is a biopharmaceutical company focused on developing orally delivered enzymes to treat disease.

Current Norton Children’s Pulmonology clinical trials and studies

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