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

NCT ID: NCT04375878

Last Updated: 2022-09-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 27 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-07-20
• Study Completion Date: 2021-04-08

Brief 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.

Detailed 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.

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

MS1819 2240 mg/day vs PERT arm,

Patients in 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.

Group Type: ACTIVE_COMPARATOR

MS1819

Intervention Type: DRUG

MS1819 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.

Porcine PERT

Intervention Type: DRUG

Porcine PERT is being used a comparator to MS1819 as a second drug/intervention.

MS1819 4480 mg/day vs PERT arm

Patients in 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.

Group Type: ACTIVE_COMPARATOR

MS1819

Intervention Type: DRUG

MS1819 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.

Porcine PERT

Intervention Type: DRUG

Porcine PERT is being used a comparator to MS1819 as a second drug/intervention.

Interventions

MS1819

MS1819 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.

Intervention Type: DRUG

Porcine PERT

Porcine PERT is being used a comparator to MS1819 as a second drug/intervention.

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Entero Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigator Site 105

Long Beach, California, United States

Investigator Site 102

Altamonte Springs, Florida, United States

Investigator Site 107

Miami, Florida, United States

Investigator Site 101

Glenview, Illinois, United States

Investigator Site 111

Wichita, Kansas, United States

Investigator Site 108

Portland, Maine, United States

Investigator Site 103

Las Vegas, Nevada, United States

Investigator Site 110

Cleveland, Ohio, United States

Investigator Site 104

Toledo, Ohio, United States

Investigator Site 106

Hershey, Pennsylvania, United States

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, , Poland

Countries

United States; Poland

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

AZ-CF2002

Identifier Type: -

Identifier Source: org_study_id