A study investigating a pancreatic enzyme therapy, not made from pigs

Full title

A phase 2 study to assess how safe and effective a new pancreatic enzyme replacement therapy, ANG003, is for people with CF and exocrine pancreatic insufficiency

Study details

Around 85-90% of people with cystic fibrosis (CF) have a condition called exocrine pancreatic insufficiency (EPI). This means their pancreas does not make any or enough digestive enzymes to help break down food. As a result, the body cannot absorb nutrients, vitamins, and energy from food as well as it should.

To help with digestion, people with CF and EPI take pancreatic enzyme replacement therapy (PERT), often called 'enzymes', whenever they eat. These enzymes help the body digest fats, proteins, and carbohydrates. Many people need to take several capsules with every meal and snack. Even when taking enzyme treatments and vitamin supplements, some people still do not absorb enough important nutrients and vitamins, especially fats.

The enzyme treatments currently available are made from pig pancreases. Over the past decade, these products have been updated to try to improve consistency and reduce potential exposure to viruses that can affect pigs and sometimes humans. There are currently shortages of the pig supply and therefore PERT in many regions around the world.

About the study treatment

This study is testing ANG003, a new enzyme treatment that is not made from pigs. Instead, it is made using highly purified enzymes produced by microorganisms. Because of how it is manufactured, ANG003 can be produced in very precise doses.

Like current enzyme treatments, ANG003 contains three types of digestive enzymes: 

• lipase, which helps digest fats
• protease, which helps digest proteins
• amylase, which helps digest carbohydrates.

The lipase in ANG003 has been designed to work more like the lipase normally produced by the pancreas, while the protease and amylase are enzymes that have already been used in other enzyme replacement treatments. The amount of each enzyme is also closer to that of a human instead of a pig as well.

Researchers want to find out how well ANG003 helps people with CF digest and absorb nutrients compared with currently available treatments.

What does taking part involve?

The study is designed in a similar way to studies that were used to approve existing enzyme replacement treatments. Researchers will assess how well food is digested and absorbed by collecting samples to measure how the body absorbs fats, proteins, and carbohydrates. This includes:

• stool (poo) samples over several days
• blood samples.

Study timeline

Before the study potential participants will first take Creon® during an Acclimation Period. This allows participants to start the study under similar conditions.

The study is then divided into two parts, Period A and Period B. The main difference between them is the enzyme treatment taken with meals.

Period A

Following the acclimation period, study participants will begin Period A. During this period study participants will be asked to do the following:

• Choose from a selection of standardised meals containing known amounts of fat, protein, and carbohydrates.
• Collect stool samples for a few days using an easy-to-use collection kit provided by the study team.
• Attend the research centre for one study day, where blood samples will be taken over several hours.

Period B

After Period A, study participants will be randomly assigned to receive either:

• one of two doses of ANG003, or
• Creon®.

During Period B, participants will:

• choose from standardised meals
• collect stool samples for a few days using the collection kit
• attend another research centre visit for blood sampling over several hours.

By comparing the results from the two study periods, researchers hope to better understand how well ANG003 helps people with CF absorb nutrients from food.
 

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