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).
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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
41
MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention
Investigator Site 105
Long Beach, California, United States
Investigator Site 102
Altamonte Springs, Florida, United States
Investigator Site 107
Miami, Florida, United States
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.
Time frame: 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
Time frame: 6 weeks
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
Number of Treatment emergent adverse events
Time frame: 6 weeks
Stool Weights
The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights
Time frame: 6 weeks
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.
Time frame: 3 weeks
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.
Time frame: 3 weeks per group.
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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
...and 4 more locations