Cystic Fibrosis (CF) Clinical Trial
— OPTIONOfficial 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
Verified date | February 2020 |
Source | First Wave Bio, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
First Wave Bio, Inc. |
United States, Poland,
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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