Trial record 2 of 2 for:    MS1819-SD

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03746483
Recruitment Status : Not yet recruiting
First Posted : November 19, 2018
Last Update Posted : November 19, 2018
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

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. 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
Cystic Fibrosis
Pancreatic enzyme replacement therapy

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