Cystic Fibrosis (CF) Clinical Trial
Official title:
OPTION 2: A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis; With an Extension Phase Evaluation of Immediate Release MS1819 Capsules
Verified date | August 2021 |
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 in enteric capsules vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). The exploratory objective of the extension phase (EP) is to find a dose of MS1819 in immediate release capsules that is safe and results in CFA values in a therapeutic range.
Status | Completed |
Enrollment | 27 |
Est. completion date | April 8, 2021 |
Est. primary completion date | April 8, 2021 |
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 either a new/historic sweat chloride =60 mmol/L (measured while not on a CFTR modulator) or genotype. 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 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 205 | Bialystok | |
Poland | Investigator Site 203 | Karpacz | |
Poland | Investigator Site 206 | Katowice | |
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 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 | Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events | Number of subjects reporting 1 or more adverse events | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) | |
Primary | Efficacy of MS1819: Coefficient of Fat Absorption (CFA) | The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. | 6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase. | |
Secondary | Stool Weights | The relative efficacy of MS1819 compared to porcine PERT will be assessed using stool weights. | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) | |
Secondary | Coefficient of Nitrogen Absorption (CNA) | CNA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. CNAs for MS1819 will be compared to the CNAs of PERT using descriptive methods. | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) |
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