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Trial record 1 of 4 for:    MS1819
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OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT04375878
Recruitment Status : Recruiting
First Posted : May 6, 2020
Last Update Posted : September 1, 2020
Sponsor:
Information provided by (Responsible Party):
AzurRx BioPharma, Inc.

Tracking Information
First Submitted Date  ICMJE April 30, 2020
First Posted Date  ICMJE May 6, 2020
Last Update Posted Date September 1, 2020
Actual Study Start Date  ICMJE July 20, 2020
Estimated Primary Completion Date December 21, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 1, 2020)
  • Safety of MS1819 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: Coefficient of fat absorption (CFA) [ Time Frame: 6 weeks ]
    The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period. CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 5, 2020)
  • Stool weights [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819 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. The signs and symptoms of malabsorption will include stool frequency, stool consistency, bloating, abdominal pain, flatulence, incidence of visible oil/grease in stool, increased stool quantity and worsening of overall bowel habit.
  • Coefficient of Nitrogen Absorption (CNA) [ Time Frame: 6 weeks ]
    CNA that will be assessed at the end of each 3-week treatment period. CNAs for MS1819 will be compared to the CNAs of PERT using descriptive methods.
Original Secondary Outcome Measures  ICMJE
 (submitted: May 1, 2020)
  • Stool weights [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819 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
  • Coefficient of Nitrogen Absorption (CNA) [ Time Frame: 6 weeks ]
    CNA that will be assessed at the end of each 3-week treatment period. CNAs for MS1819 will be compared to the CNAs of PERT using descriptive methods.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic Fibrosis
Official Title  ICMJE 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
Brief Summary 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).
Detailed Description

This is a Phase 2, open-label, multicenter, 2, 2x2 crossover study assessing the safety and efficacy of MS1819, 2240 mg/day vs porcine PERT, and 4480 mg/day vs porcine PERT given at the same dose and dosing regimen that was being administered during the pre-study period. MS1819 will be administered in enteric capsules.

MS1819 will be assessed in a 2x2 crossover including approximately 30 patients completing both periods. Fifteen patients will be randomized to the MS1819 2240 mg/day vs PERT arm, and 15 patients will be randomized to the MS1819 4480 mg/day vs PERT arm. Patients in each arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.The coefficient of fat absorption (CFA) will be measured at the end of each 3 week study period.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
2, 2x2 Crossover
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Exocrine Pancreatic Insufficiency (EPI)
  • Cystic Fibrosis (CF)
Intervention  ICMJE
  • Drug: MS1819
    MS1819 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
  • Drug: Porcine PERT
    Porcine PERT is being used a comparator to MS1819 as a second drug/intervention.
Study Arms  ICMJE
  • Active Comparator: MS1819 2240 mg/day vs PERT arm,
    Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.
    Interventions:
    • Drug: MS1819
    • Drug: Porcine PERT
  • Active Comparator: MS1819 4480 mg/day vs PERT arm
    Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.
    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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 1, 2020)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 22, 2021
Estimated Primary Completion Date December 21, 2020   (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 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
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: Monica Gangal 646-699-7855 mgangal@azurrx.com
Listed Location Countries  ICMJE Poland,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04375878
Other Study ID Numbers  ICMJE AZ-CF2002
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
Responsible Party AzurRx BioPharma, Inc.
Study Sponsor  ICMJE AzurRx BioPharma, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account AzurRx BioPharma, Inc.
Verification Date August 2020

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