OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

• Status: Completed
• Trial end date: 2019-07-27
• Target enrollment: 0
• Participant gender: All

Summary

The primary objectives of this study are to assess the safety and efficacy of MS1819-SD vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).

• 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 initial diagnostic sweat chloride ≥ 60 mmol/L

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