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

Cystic Fibrosis (CF) Clinical Trial

Official title:

OPTION 2: A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis; With an Extension Phase Evaluation of Immediate Release MS1819 Capsules

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04375878
• Other study ID #: AZ-CF2002
• Status: Completed
• Phase: Phase 2
• Start date: July 20, 2020
• Est. completion date: April 8, 2021

Study information

• Verified date: August 2021
• 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 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.

Description:

This is a Phase 2, open-label, multicenter, 2, 2x2 crossover study assessing the safety and efficacy of MS1819, 2240 mg/day vs porcine PERT, and 4480 mg/day vs porcine PERT given at the same dose and dosing regimen that was being administered during the pre-study period. MS1819 will be administered in enteric capsules. MS1819 will be assessed in a 2x2 crossover including approximately 30 patients completing both periods. Fifteen patients will be randomized to the MS1819 2240 mg/day vs PERT arm, and 15 patients will be randomized to the MS1819 4480 mg/day vs PERT arm. Patients in each arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.The coefficient of fat absorption (CFA) will be measured at the end of each 3 week study period. Patients enrolled into the extension phase (EP) will be composed of patients who have completed the crossover phase of OPTION 2.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: April 8, 2021
• Est. primary completion date: April 8, 2021
• 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 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

Related Conditions & MeSH terms

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

Intervention

Drug:
• MS1819
MS1819 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
• Porcine PERT
Porcine PERT is being used a comparator to MS1819 as a second drug/intervention.

Locations

Country	Name	City	State
Poland	Investigator Site 205	Bialystok
Poland	Investigator Site 203	Karpacz
Poland	Investigator Site 206	Katowice
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 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	Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events	Number of subjects reporting 1 or more adverse events	Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Primary	Efficacy of MS1819: Coefficient of Fat Absorption (CFA)	The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods.	6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase.
Secondary	Stool Weights	The relative efficacy of MS1819 compared to porcine PERT will be assessed using stool weights.	Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Secondary	Coefficient of Nitrogen Absorption (CNA)	CNA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. CNAs for MS1819 will be compared to the CNAs of PERT using descriptive methods.	Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)