Clinical trial NCT04375878: 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; With an Extension Phase Evaluation of Immediate Release MS1819 Capsules
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Exocrine Pancreatic Insufficiency (EPI)
Cystic Fibrosis (CF)
Keywords: Exocrine Pancreatic Insufficiency
EPI
Cystic Fibrosis
CF
Lead sponsor: Entero Therapeutics
Sponsor class: INDUSTRY
First posted: 2020-05-06
Enrollment: 27
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: 2, 2x2 Crossover
Primary purpose: TREATMENT

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). The exploratory objective of the extension phase (EP) is to find a dose of MS1819 in immediate release capsules that is safe and results in CFA values in a therapeutic range.

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. Patients enrolled into the extension phase (EP) will be composed of patients who have completed the crossover phase of OPTION 2.

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

Primary outcomes:
measure: Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events
timeFrame: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
measure: Efficacy of MS1819: Coefficient of Fat Absorption (CFA)
timeFrame: 6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase.
Secondary outcomes:
measure: Stool Weights
timeFrame: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
measure: Coefficient of Nitrogen Absorption (CNA)
timeFrame: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Other outcomes:
Locations:
city: Long Beach
country: United States
facility: Investigator Site 105
state: California
city: Altamonte Springs
country: United States
facility: Investigator Site 102
state: Florida
city: Miami
country: United States
facility: Investigator Site 107
state: Florida
city: Glenview
country: United States
facility: Investigator Site 101
state: Illinois
city: Wichita
country: United States
facility: Investigator Site 111
state: Kansas
city: Portland
country: United States
facility: Investigator Site 108
state: Maine
city: Las Vegas
country: United States
facility: Investigator Site 103
state: Nevada
city: Cleveland
country: United States
facility: Investigator Site 110
state: Ohio
city: Toledo
country: United States
facility: Investigator Site 104
state: Ohio
city: Hershey
country: United States
facility: Investigator Site 106
state: Pennsylvania
city: Białystok
country: Poland
facility: Investigator Site 205
city: Karpacz
country: Poland
facility: Investigator Site 203
city: Katowice
country: Poland
facility: Investigator Site 206
city: Rabka-Zdrój
country: Poland
facility: Investigator Site 202
city: Sopot
country: Poland
facility: Investigator Site 204
Study officials:
References: