We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis (OPTION)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03746483
Recruitment Status : Completed
First Posted : November 19, 2018
Results First Posted : June 6, 2022
Last Update Posted : June 6, 2022
Sponsor:
Information provided by (Responsible Party):
First Wave Bio, Inc.

Tracking Information
First Submitted Date  ICMJE November 15, 2018
First Posted Date  ICMJE November 19, 2018
Results First Submitted Date  ICMJE March 4, 2022
Results First Posted Date  ICMJE June 6, 2022
Last Update Posted Date June 6, 2022
Actual Study Start Date  ICMJE January 10, 2019
Actual Primary Completion Date July 13, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 12, 2022)
  • Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA) [ Time Frame: 3 weeks ]
    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) [ Time Frame: 6 weeks ]
    Number of participants reporting 1 or more adverse events
  • Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 6 weeks ]
    Number of Treatment emergent adverse events
Original Primary Outcome Measures  ICMJE
 (submitted: November 16, 2018)
  • Safety of MS1819-SD by number of subjects reporting 1 or more adverse events [ Time Frame: 6 weeks ]
    Number of subjects reporting 1 or more adverse events
  • Efficacy of MS1819-SD: Coefficient of fat absorption (CFA) [ Time Frame: 6 weeks ]
    Coefficient of fat absorption (CFA)
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 4, 2022)
  • Stool Weights [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights
  • Signs and Symptoms of Malabsorption [ Time Frame: 3 weeks ]
    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) [ Time Frame: 3 weeks per group. ]
    CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.
Original Secondary Outcome Measures  ICMJE
 (submitted: November 16, 2018)
  • Stool weights [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights
  • Signs and symptoms of malabsorption [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819 compared to porcine PERT will be assessed using signs and symptoms of malabsorption
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
Official Title  ICMJE 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
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
2x2 Crossover
Masking: None (Open Label)
Masking Description:
Unblinded
Primary Purpose: Treatment
Condition  ICMJE
  • Exocrine Pancreatic Insufficiency (EPI)
  • Cystic Fibrosis (CF)
Intervention  ICMJE
  • Drug: MS1819
    MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
  • Drug: Porcine PERT
    Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention
Study Arms  ICMJE
  • Experimental: MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks)
    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.
    Interventions:
    • Drug: MS1819
    • Drug: Porcine PERT
  • Experimental: PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)
    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.
    Interventions:
    • Drug: MS1819
    • Drug: Porcine PERT
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 4, 2022)
41
Original Estimated Enrollment  ICMJE
 (submitted: November 16, 2018)
30
Actual Study Completion Date  ICMJE July 27, 2019
Actual Primary Completion Date July 13, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Poland,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03746483
Other Study ID Numbers  ICMJE AZ-CF2001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party First Wave Bio, Inc.
Original Responsible Party AzurRx BioPharma, Inc.
Current Study Sponsor  ICMJE First Wave Bio, Inc.
Original Study Sponsor  ICMJE AzurRx BioPharma, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account First Wave Bio, Inc.
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP