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Phase 2 N=41 Randomized Treatment

OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

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

Enrolled (actual)
41
Serious AEs
0.0%
Results posted
Jun 2022
Primary outcome: Primary: Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA) — 55.6; 86.2 % CFA

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
MS1819 (Drug); Porcine PERT (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Entero Therapeutics
Primary completion
Jul 2019

Outcome Measures

OutcomeResultp-value
PRIMARY
Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)
55.6; 86.2
PRIMARY
Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)
13; 6; 2; 0
PRIMARY
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
19; 8; 0; 0
SECONDARY
Stool Weights
1394.0; 727.3
SECONDARY
Signs and Symptoms of Malabsorption
15; 32; 12; 3; 6; 1
SECONDARY
Coefficient of Nitrogen Absorption (CNA)
93.0; 97.2

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).

Eligibility Criteria

Inclusion Criteria

  • Cystic fibrosis, based on 2 clinical features consistent with CF, plus initial diagnostic sweat chloride ≥ 60 mmol/L
  • Under stable dose of porcine PERT
  • A fair or better nutritional status
  • Fecal elastase <100 µg/g
  • Standard-of-care medications including Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators are allowed

Exclusion Criteria

  • History or diagnosis of fibrosing colonopathy
  • Any chronic diarrheal illness unrelated to pancreatic insufficiency
  • 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
  • Feeding via an enteral tube during 6 months before screening
  • Forced expiratory volume ≤30% at the Screening visit
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03746483). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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