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NCT Number: NCT03746483

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

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).

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Key information

Conditions

Exocrine Pancreatic Insufficiency (EPI) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Cystic Fibrosis (CF) Digestive System Diseases Exocrine Pancreatic Insufficiency Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Investigator Site 203, Karpacz, Poland

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

MS1819

Drug

MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.

Porcine PERT

Drug

Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention

Primary outcomes

  1. Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)

    Time frame: 3 weeks

    The Coefficient of Fat Absorption (CFA%) is defined as:

    [72-hour fat intake (g) - 72-hour fat excretion (g)/72-hour fat intake(g)] x 100 = CFA% The threshold for CFA results (>80%) is considered clinically significant for treatment effectiveness by the FDA.

  2. Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)

    Time frame: 6 weeks

    Number of participants reporting 1 or more adverse events

  3. Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)

    Time frame: 6 weeks

    Number of Treatment emergent adverse events

Secondary outcomes

  1. Stool Weights

    Time frame: 6 weeks

    The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights

  2. Signs and Symptoms of Malabsorption

    Time frame: 3 weeks

    The relative efficacy of MS1819 compared to porcine PERT will be assessed using signs and symptoms of malabsorption. Abdominal pain, bloating, flatulence, increased stool quantity, and worsening of overall bowel habit were graded as 0 = none, 1 = mild, 2 = moderate, or 3 = severe.

  3. Coefficient of Nitrogen Absorption (CNA)

    Time frame: 3 weeks per group.

    CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.

Sponsors and collaborators

Lead sponsor

Entero Therapeutics

Industry

Registry information

Official study 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

Acronym: OPTION

Important dates

Study start
2019
Primary completion
2019
Study completion
2019
First posted
Nov 19, 2018
Registry last updated
Jun 6, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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