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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03746483
Recruitment Status : Completed
First Posted : November 19, 2018
Results First Posted : June 6, 2022
Last Update Posted : June 6, 2022
Sponsor:
Information provided by (Responsible Party):
First Wave BioPharma, Inc.

Study Description
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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).

Condition or disease Intervention/treatment Phase
Exocrine Pancreatic Insufficiency (EPI) Cystic Fibrosis (CF) Drug: MS1819 Drug: Porcine PERT Phase 2

Detailed 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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: 2x2 Crossover
Masking: None (Open Label)
Masking Description: Unblinded
Primary Purpose: Treatment
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
Actual Study Start Date : January 10, 2019
Actual Primary Completion Date : July 13, 2019
Actual Study Completion Date : July 27, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks)
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
MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.

Drug: Porcine PERT
Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention
Experimental: PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)
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
MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.

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


Outcome Measures
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Primary Outcome Measures :
  1. Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA) [ Time Frame: 3 weeks ]

    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.


  2. Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE) [ Time Frame: 6 weeks ]
    Number of participants reporting 1 or more adverse events

  3. Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 6 weeks ]
    Number of Treatment emergent adverse events


Secondary Outcome Measures :
  1. Stool Weights [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights

  2. Signs and Symptoms of Malabsorption [ Time Frame: 3 weeks ]
    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. Coefficient of Nitrogen Absorption (CNA) [ Time Frame: 3 weeks per group. ]
    CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03746483


Locations
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United States, California
Investigator Site 105
Long Beach, California, United States, 90806
United States, Florida
Investigator Site 102
Altamonte Springs, Florida, United States, 32701
Investigator Site 107
Miami, Florida, United States, 33136
United States, Illinois
Investigator Site 101
Glenview, Illinois, United States, 60025
United States, Kansas
Investigator Site 111
Wichita, Kansas, United States, 67214
United States, Maine
Investigator Site 108
Portland, Maine, United States, 04102
United States, Nevada
Investigator Site 103
Las Vegas, Nevada, United States, 89109
United States, Ohio
Investigator Site 110
Cleveland, Ohio, United States, 44106
Investigator Site 104
Toledo, Ohio, United States, 43606
United States, Pennsylvania
Investigator Site 106
Hershey, Pennsylvania, United States, 17033
United States, Texas
Investigator Site 109
Dallas, Texas, United States, 75235
Poland
Investigator Site 203
Karpacz, Poland
Investigator Site 202
Rabka-Zdrój, Poland
Investigator Site 204
Sopot, Poland
Sponsors and Collaborators
First Wave BioPharma, Inc.
  Study Documents (Full-Text)

Documents provided by First Wave BioPharma, Inc.:
Study Protocol  [PDF] October 30, 2018
Statistical Analysis Plan  [PDF] September 4, 2019

More Information
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Responsible Party: First Wave BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT03746483    
Other Study ID Numbers: AZ-CF2001
First Posted: November 19, 2018    Key Record Dates
Results First Posted: June 6, 2022
Last Update Posted: June 6, 2022
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by First Wave BioPharma, Inc.:
Exocrine pancreatic insufficiency
EPI
Cystic Fibrosis
 CF
Pancreatic enzyme replacement therapy (PERT)
Lipase
Additional relevant MeSH terms:
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Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Fibrosis
Pathologic Processes
Pancreatic Diseases
 Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases