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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 : Not yet recruiting
First Posted : November 19, 2018
Last Update Posted : November 19, 2018
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.


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
Estimated Study Start Date : December 20, 2018
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

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: November 19, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
Fibrosis
Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases