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

Cystic Fibrosis (CF) Clinical Trial

— OPTION  

Official title:

A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819-SD in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03746483
• Other study ID #: AZ-CF2001
• Status: Completed
• Phase: Phase 2
• Start date: January 10, 2019
• Est. completion date: July 27, 2019

Study information

• Verified date: February 2020
• Source: First Wave Bio, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Description:

This is a Phase 2, open-label, multi-center, 2x2 crossover study assessing the safety and efficacy of MS1819-SD (spray dried) vs porcine PERT given at the same dose that was being administered during the pre-study period. MS1819-SD will be assessed in a 2x2 crossover including at least 30 patients completing both periods.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 41
• Est. completion date: July 27, 2019
• Est. primary completion date: July 13, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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 Cystic Fibrosis Transmembrane Conductance Regulator (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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Cystic Fibrosis (CF)
• Exocrine Pancreatic Insufficiency
• Exocrine Pancreatic Insufficiency (EPI)
• Fibrosis

Intervention

Drug:
• MS1819
MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
• Porcine PERT
Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention

Locations

Country	Name	City	State
Poland	Investigator Site 203	Karpacz
Poland	Investigator Site 202	Rabka-Zdrój
Poland	Investigator Site 204	Sopot
United States	Investigator Site 102	Altamonte Springs	Florida
United States	Investigator Site 110	Cleveland	Ohio
United States	Investigator Site 109	Dallas	Texas
United States	Investigator Site 101	Glenview	Illinois
United States	Investigator Site 106	Hershey	Pennsylvania
United States	Investigator Site 103	Las Vegas	Nevada
United States	Investigator Site 105	Long Beach	California
United States	Investigator Site 107	Miami	Florida
United States	Investigator Site 108	Portland	Maine
United States	Investigator Site 104	Toledo	Ohio
United States	Investigator Site 111	Wichita	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
First Wave Bio, Inc.

Countries where clinical trial is conducted

United States,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)	The Coefficient of Fat Absorption (CFA%) is defined as: [72-hour fat intake (g) - 72-hour fat excretion (g)/72-hour fat intake(g)] x 100 = CFA% The threshold for CFA results (>80%) is considered clinically significant for treatment effectiveness by the FDA.	3 weeks
Primary	Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)	Number of participants reporting 1 or more adverse events	6 weeks
Primary	Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)	Number of Treatment emergent adverse events	6 weeks
Secondary	Stool Weights	The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights	6 weeks
Secondary	Signs and Symptoms of Malabsorption	The relative efficacy of MS1819 compared to porcine PERT will be assessed using signs and symptoms of malabsorption. Abdominal pain, bloating, flatulence, increased stool quantity, and worsening of overall bowel habit were graded as 0 = none, 1 = mild, 2 = moderate, or 3 = severe.	3 weeks
Secondary	Coefficient of Nitrogen Absorption (CNA)	CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.	3 weeks per group.