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

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

Tracking Information

• First Submitted Date: April 30, 2020
• First Posted Date: May 6, 2020
• Last Update Posted Date: September 1, 2020
• Actual Study Start Date: July 20, 2020
• Estimated Primary Completion Date: December 21, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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: Same as current

Current Secondary Outcome Measures (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 (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: OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic Fibrosis
• 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
• 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: Interventional
• Study Phase: Phase 2

Study Design

Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:

2, 2x2 Crossover

Masking: None (Open Label)
Primary Purpose: Treatment

Condition

• Exocrine Pancreatic Insufficiency (EPI)
• Cystic Fibrosis (CF)

Intervention

• 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

• 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

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: May 1, 2020): 30
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: February 22, 2021
• Estimated Primary Completion Date: December 21, 2020 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: Poland, United States

Administrative Information

• NCT Number: NCT04375878
• Other Study ID Numbers: 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

• Plan to Share IPD: No

• Responsible Party: AzurRx BioPharma, Inc.
• Study Sponsor: AzurRx BioPharma, Inc.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: AzurRx BioPharma, Inc.
• Verification Date: August 2020