OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic Fibrosis

• Status: Completed
• Trial end date: 2021-04-08
• Target enrollment: 0
• Participant gender: All

Summary

The primary objectives of this study are to assess the safety and efficacy of MS1819 in enteric capsules vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). The exploratory objective of the extension phase (EP) is to find a dose of MS1819 in immediate release capsules that is safe and results in CFA values in a therapeutic range.

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

Details

• Lead Sponsor: AzurRx BioPharma, Inc.

Criteria

Inclusion Criteria:

1. Cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride ≥60 mmol/L (measured while not on a CFTR modulator) or genotype.

2. Under stable dose of porcine PERT

3. A fair or better nutritional status

4. Fecal elastase <100 µg/g

5. 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 the Screening visit

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

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