Back to resultsOPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis (OPTION)
Primary Purpose
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
MS1819
Porcine PERT
Sponsored by
About this trial
This is an interventional treatment trial for Exocrine Pancreatic Insufficiency (EPI) focused on measuring Exocrine pancreatic insufficiency, EPI, Cystic Fibrosis, CF, Pancreatic enzyme replacement therapy (PERT), Lipase
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Sites / Locations
- Investigator Site 105
- Investigator Site 102
- Investigator Site 107
- Investigator Site 101
- Investigator Site 111
- Investigator Site 108
- Investigator Site 103
- Investigator Site 110
- Investigator Site 104
- Investigator Site 106
- Investigator Site 109
- Investigator Site 203
- Investigator Site 202
- Investigator Site 204
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
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)
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
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.
Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)
Number of participants reporting 1 or more adverse events
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
Number of Treatment emergent adverse events
Secondary Outcome Measures
Stool Weights
The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights
Signs and Symptoms of Malabsorption
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.
Coefficient of Nitrogen Absorption (CNA)
CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.
Full Information
NCT ID
NCT03746483
First Posted
November 15, 2018
Last Updated
May 12, 2022
Sponsor
First Wave BioPharma, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03746483
Brief Title
OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
Acronym
OPTION
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2020
Overall Recruitment Status
Completed
Study Start Date
January 10, 2019 (Actual)
Primary Completion Date
July 13, 2019 (Actual)
Study Completion Date
July 27, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
First Wave BioPharma, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Keywords
Exocrine pancreatic insufficiency, EPI, Cystic Fibrosis, CF, Pancreatic enzyme replacement therapy (PERT), Lipase
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Model Description
2x2 Crossover
Masking
None (Open Label)
Masking Description
Unblinded
Allocation
Randomized
Enrollment
41 (Actual)
8. Arms, Groups, and Interventions
Arm Title
MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks)
Arm Type
Experimental
Arm Description
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.
Arm Title
PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
MS1819
Intervention Description
MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
Intervention Type
Drug
Intervention Name(s)
Porcine PERT
Intervention Description
Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention
Primary Outcome Measure Information:
Title
Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)
Description
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.
Time Frame
3 weeks
Title
Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)
Description
Number of participants reporting 1 or more adverse events
Time Frame
6 weeks
Title
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
Description
Number of Treatment emergent adverse events
Time Frame
6 weeks
Secondary Outcome Measure Information:
Title
Stool Weights
Description
The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights
Time Frame
6 weeks
Title
Signs and Symptoms of Malabsorption
Description
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.
Time Frame
3 weeks
Title
Coefficient of Nitrogen Absorption (CNA)
Description
CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.
Time Frame
3 weeks per group.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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
Facility Information:
Facility Name
Investigator Site 105
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
Investigator Site 102
City
Altamonte Springs
State/Province
Florida
ZIP/Postal Code
32701
Country
United States
Facility Name
Investigator Site 107
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Investigator Site 101
City
Glenview
State/Province
Illinois
ZIP/Postal Code
60025
Country
United States
Facility Name
Investigator Site 111
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67214
Country
United States
Facility Name
Investigator Site 108
City
Portland
State/Province
Maine
ZIP/Postal Code
04102
Country
United States
Facility Name
Investigator Site 103
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89109
Country
United States
Facility Name
Investigator Site 110
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Investigator Site 104
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Facility Name
Investigator Site 106
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Investigator Site 109
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Investigator Site 203
City
Karpacz
Country
Poland
Facility Name
Investigator Site 202
City
Rabka-Zdrój
Country
Poland
Facility Name
Investigator Site 204
City
Sopot
Country
Poland
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
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