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

NCT04375878 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: AZ-CF2002
• Study Type: interventional
• Target: 27
• Locations: 2 countries
• Sites: 15

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.

Conditions

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

Keywords

Exocrine Pancreatic Insufficiency; EPI; Cystic Fibrosis; CF

Outcome Measures

Primary Outcomes (2)

• 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)

• 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 Outcomes (2)

• Stool Weights

  Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)

• Coefficient of Nitrogen Absorption (CNA)

  Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)

Study Arms (2)

MS1819 2240 mg/day vs PERT arm,

ACTIVE COMPARATOR

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.

Drug: MS1819; Drug: Porcine PERT

MS1819 4480 mg/day vs PERT arm

ACTIVE COMPARATOR

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.

Drug: MS1819; Drug: Porcine PERT

Interventions

MS1819 DRUG

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

MS1819 2240 mg/day vs PERT arm,; MS1819 4480 mg/day vs PERT arm

Porcine PERT DRUG

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

MS1819 2240 mg/day vs PERT arm,; MS1819 4480 mg/day vs PERT arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Under stable dose of porcine PERT
• A fair or better nutritional status
• Fecal elastase \<100 µg/g
• Standard-of-care medications including CFTR modulators are allowed

You may not qualify if:

• History or diagnosis of fibrosing colonopathy
• Any chronic diarrheal illness unrelated to pancreatic insufficiency
• 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
• Feeding via an enteral tube during 6 months before screening
• Forced expiratory volume ≤30% at the Screening visit

Sponsors & Collaborators

• Entero Therapeutics: lead

Study Sites (15)

Investigator Site 105

Long Beach, California, 90806, United States

Location

Investigator Site 102

Altamonte Springs, Florida, 32701, United States

Location

Investigator Site 107

Miami, Florida, 33136, United States

Location

Investigator Site 101

Glenview, Illinois, 60025, United States

Location

Investigator Site 111

Wichita, Kansas, 67214, United States

Location

Investigator Site 108

Portland, Maine, 04102, United States

Location

Investigator Site 103

Las Vegas, Nevada, 89109, United States

Location

Investigator Site 110

Cleveland, Ohio, 44106, United States

Location

Investigator Site 104

Toledo, Ohio, 43606, United States

Location

Investigator Site 106

Hershey, Pennsylvania, 17033, United States

Location

Investigator Site 205

Bialystok, Poland

Location

Investigator Site 203

Karpacz, Poland

Location

Investigator Site 206

Katowice, Poland

Location

Investigator Site 202

Rabka-Zdrój, Poland

Location

Investigator Site 204

Sopot, Poland

Location

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Chief Medical Officer
• Organization: First Wave BioPharma Inc.

info@firstwavebio.com

(561) 589 7020

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 2, 2x2 Crossover

Study Record Dates

• First Submitted: April 30, 2020
• First Posted: May 6, 2020
• Study Start: July 20, 2020
• Primary Completion: April 8, 2021
• Study Completion: April 8, 2021
• Last Updated: September 13, 2022
• Results First Posted: September 13, 2022

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

PL (5); US (10)