Clinical trial NCT03746483: OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Exocrine Pancreatic Insufficiency (EPI)
Cystic Fibrosis (CF)
Keywords: Exocrine pancreatic insufficiency
EPI
Cystic Fibrosis
CF
Pancreatic enzyme replacement therapy (PERT)
Lipase
Lead sponsor: Entero Therapeutics
Sponsor class: INDUSTRY
First posted: 2018-11-19
Enrollment: 41
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: 2x2 Crossover
Primary purpose: TREATMENT

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).

Detailed description: This is a Phase 2, open-label, multi-center, 2x2 crossover study assessing the safety and efficacy of MS1819-SD (spray dried) 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.

Eligibility 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 Cystic Fibrosis Transmembrane Conductance Regulator (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: Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)
timeFrame: 3 weeks
measure: Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)
timeFrame: 6 weeks
measure: Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
timeFrame: 6 weeks
Secondary outcomes:
measure: Stool Weights
timeFrame: 6 weeks
measure: Signs and Symptoms of Malabsorption
timeFrame: 3 weeks
measure: Coefficient of Nitrogen Absorption (CNA)
timeFrame: 3 weeks per group.
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: Dallas
country: United States
facility: Investigator Site 109
state: Texas
city: Karpacz
country: Poland
facility: Investigator Site 203
city: Rabka-Zdrój
country: Poland
facility: Investigator Site 202
city: Sopot
country: Poland
facility: Investigator Site 204
Study officials:
References: