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

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.
 
ClinicalTrials.gov Identifier: NCT04375878
Recruitment Status : Recruiting
First Posted : May 6, 2020
Last Update Posted : July 24, 2020
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 in enteric capsules 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 Drug: Porcine PERT Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: 2, 2x2 Crossover
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : July 20, 2020
Estimated Primary Completion Date : December 21, 2020
Estimated Study Completion Date : February 22, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
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.
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.

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.
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.




Primary Outcome Measures :
  1. 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

  2. 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.


Secondary Outcome Measures :
  1. Stool weights [ Time Frame: 6 weeks ]
    The relative efficacy of MS1819 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. 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.

  3. 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.



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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 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

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): NCT04375878


Contacts
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Contact: Monica Gangal 646-699-7855 mgangal@azurrx.com

Locations
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United States, Florida
Investigator Site 102 Recruiting
Altamonte Springs, Florida, United States, 32701
United States, Illinois
Investigator Site 101 Recruiting
Glenview, Illinois, United States, 60025
United States, Nevada
Investigator Site 103 Recruiting
Las Vegas, Nevada, United States, 89109
United States, Ohio
Investigator Site 104 Recruiting
Toledo, Ohio, United States, 43606
Sponsors and Collaborators
AzurRx BioPharma, Inc.
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Responsible Party: AzurRx BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT04375878    
Other Study ID Numbers: AZ-CF2002
First Posted: May 6, 2020    Key Record Dates
Last Update Posted: July 24, 2020
Last Verified: May 2020
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
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