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

NCT03746483 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: AZ-CF2001
• Study Type: interventional
• Target: 41
• Locations: 2 countries
• Sites: 14

Brief 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).

Conditions

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

Keywords

Exocrine pancreatic insufficiency; EPI; Cystic Fibrosis; CF; Pancreatic enzyme replacement therapy (PERT); Lipase

Outcome Measures

Primary Outcomes (3)

• 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

• 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

• Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)

  Number of Treatment emergent adverse events

  6 weeks

Secondary Outcomes (3)

• Stool Weights

  6 weeks

• Signs and Symptoms of Malabsorption

  3 weeks

• Coefficient of Nitrogen Absorption (CNA)

  3 weeks per group.

Study Arms (2)

MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks)

EXPERIMENTAL

Patients in arm will further be randomized to receive either the sequence consisting of MS1819 2240 mg/day for 3 weeks followed by PERT for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.

Drug: MS1819; Drug: Porcine PERT

PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)

EXPERIMENTAL

Patients in arm will be randomized to receive PERT for 3 weeks followed by MS1819 2240 mg/day for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.

Drug: MS1819; Drug: Porcine PERT

Interventions

MS1819 DRUG

MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.

MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks); PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)

Porcine PERT DRUG

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

MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks); PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)

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 initial diagnostic sweat chloride ≥ 60 mmol/L
• Under stable dose of porcine PERT
• A fair or better nutritional status
• Fecal elastase \<100 µg/g
• Standard-of-care medications including Cystic Fibrosis Transmembrane Conductance Regulator (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 (14)

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 109

Dallas, Texas, 75235, United States

Location

Investigator Site 203

Karpacz, 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
• Masking Details: Unblinded
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 2x2 Crossover

Study Record Dates

• First Submitted: November 15, 2018
• First Posted: November 19, 2018
• Study Start: January 10, 2019
• Primary Completion: July 13, 2019
• Study Completion: July 27, 2019
• Last Updated: June 6, 2022
• Results First Posted: June 6, 2022

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will not share

Locations

PL (3); US (11)