Pilot Study to Assess Safety and Efficacy of an Enteric Microgranule Formulation of Adrulipase in Patients With Exocrine Pancreatic Insufficiency (EPI) Due to Cystic Fibrosis (CF).

• Status: Recruiting
• Trial end date: 2023-06-01
• Target enrollment: 0
• Participant gender: All

Summary

Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI). Currently, these patients take pancreatic enzymes that are obtained from pig pancreas to aid the digestion of food. The goals of this clinical study are to evaluate the safety and efficacy of a novel formulation of a non-porcine lipase, called adrulipase, in patients with EPI due to cystic fibrosis.

The main question[s] the study aims to answer are:

1. Is the novel formulation of adrulipase safe to use at the doses being evaluated in the clinical study.

2. Is adrulipase as effective, or more effective, compared to the pig enzymes the patients currently use.

Researchers will compare the results obtained with adrulipase to how the patients typically respond to their pig enzymes to see if adrulipase helps patients digest fats adequately and if their stomach feels good (signs and symptoms of malabsorption).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: First Wave Bio, Inc.

Criteria

Inclusion Criteria:

1. A confirmed diagnosis of cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride >60 mmol/L by quantitative pilocarpine iontophoresis (measured while not on a CFTR modulator) or genotype.

2. On stable dose of porcine PERT ≥1 month (30 days) prior to screening; stable dose is defined as dose of medication not changed during this time period, and the medication must be commercially available and be administered in the recommended dose range.

3. CFA = or > 80% at screening while on stable PERT

4. A fair or better nutritional status as defined by:

• BMI ≥16.0 kg/m2 for female patients ≥18 years of age, or

• BMI ≥16.5 kg/m2 for male patients ≥18 years of age

5. Fecal elastase <100 µg/g of stool at screening

6. Standard-of-Care medications including CFTR modulators are allowed

Exclusion Criteria:

1. History or diagnosis of fibrosing colonopathy

2. Any chronic diarrheal illness unrelated to pancreatic insufficiency

3. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level

• 5 ×upper limit of normal (ULN), or total bilirubin level ≥1.5 ×ULN at Screening

4. Feeding via an enteral tube during 6 months before screening

5. Forced expiratory volume ≤30% at the Screening visit

6. Changes in gastric acid suppressant therapy during the one month prior to screening for patients already on suppressant therapy.