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Trial record 2 of 2 for:    ms1819

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

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ClinicalTrials.gov Identifier: NCT03746483
Recruitment Status : Recruiting
First Posted : November 19, 2018
Last Update Posted : May 8, 2019
Sponsor:
Information provided by (Responsible Party):
AzurRx BioPharma, Inc.

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).

Condition or disease Intervention/treatment Phase
Exocrine Pancreatic Insufficiency (EPI) Cystic Fibrosis (CF) Drug: MS1819-SD Drug: Porcine PERT Phase 2

Detailed Description:

This is a Phase 2, open-label, multi-center, 2x2 crossover study assessing the safety and efficacy of MS1819-SD 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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: 2x2 Crossover
Masking: None (Open Label)
Masking Description: Unblinded
Primary Purpose: Treatment
Official 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
Actual Study Start Date : January 10, 2019
Estimated Primary Completion Date : June 30, 2019
Estimated Study Completion Date : August 31, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis


Intervention Details:
  • Drug: MS1819-SD
    MS1819-SD, 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-SD as a second drug/intervention


Primary Outcome Measures :
  1. 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

  2. Efficacy of MS1819-SD: Coefficient of fat absorption (CFA) [ Time Frame: 6 weeks ]
    Coefficient of fat absorption (CFA)


Secondary Outcome Measures :
  1. Stool weights [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights

  2. 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



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03746483


Contacts
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Contact: Monica Gangal 6466997855 mgangal@azurrx.com

Locations
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United States, California
Investigator Site 105 Recruiting
Long Beach, California, United States, 90806
United States, Florida
Investigator Site 102 Recruiting
Altamonte Springs, Florida, United States, 32701
Investigator Site 107 Recruiting
Miami, Florida, United States, 33136
United States, Illinois
Investigator Site 101 Recruiting
Glenview, Illinois, United States, 60025
United States, Kansas
Investigator Site 111 Recruiting
Wichita, Kansas, United States, 67214
United States, Maine
Investigator Site 108 Recruiting
Portland, Maine, United States, 04102
United States, Nevada
Investigator Site 103 Recruiting
Las Vegas, Nevada, United States, 89109
United States, Ohio
Investigator Site 110 Recruiting
Cleveland, Ohio, United States, 44106
Investigator Site 104 Recruiting
Toledo, Ohio, United States, 43606
United States, Pennsylvania
Investigator Site 106 Recruiting
Hershey, Pennsylvania, United States, 17033
United States, Texas
Investigator Site 109 Recruiting
Dallas, Texas, United States, 75235
Poland
Investigator Site 203 Recruiting
Karpacz, Poland
Investigator Site 202 Recruiting
Rabka-Zdrój, Poland
Investigator Site 204 Recruiting
Sopot, Poland
Sponsors and Collaborators
AzurRx BioPharma, Inc.

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Responsible Party: AzurRx BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT03746483     History of Changes
Other Study ID Numbers: AZ-CF2001
First Posted: November 19, 2018    Key Record Dates
Last Update Posted: May 8, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by AzurRx BioPharma, Inc.:
Exocrine pancreatic insufficiency
EPI
Cystic Fibrosis
CF
Pancreatic enzyme replacement therapy
Lipase
Additional relevant MeSH terms:
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Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases