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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03746483
Other study ID # AZ-CF2001
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date January 10, 2019
Est. completion date July 27, 2019

Study information

Verified date February 2020
Source First Wave Bio, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


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.


Recruitment information / eligibility

Status Completed
Enrollment 41
Est. completion date July 27, 2019
Est. primary completion date July 13, 2019
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
MS1819
MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
Porcine PERT
Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention

Locations

Country Name City State
Poland Investigator Site 203 Karpacz
Poland Investigator Site 202 Rabka-Zdrój
Poland Investigator Site 204 Sopot
United States Investigator Site 102 Altamonte Springs Florida
United States Investigator Site 110 Cleveland Ohio
United States Investigator Site 109 Dallas Texas
United States Investigator Site 101 Glenview Illinois
United States Investigator Site 106 Hershey Pennsylvania
United States Investigator Site 103 Las Vegas Nevada
United States Investigator Site 105 Long Beach California
United States Investigator Site 107 Miami Florida
United States Investigator Site 108 Portland Maine
United States Investigator Site 104 Toledo Ohio
United States Investigator Site 111 Wichita Kansas

Sponsors (1)

Lead Sponsor Collaborator
First Wave Bio, Inc.

Countries where clinical trial is conducted

United States,  Poland, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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
Primary 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
Primary Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs) Number of Treatment emergent adverse events 6 weeks
Secondary Stool Weights The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights 6 weeks
Secondary 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. 3 weeks
Secondary 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. 3 weeks per group.
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