Phase 2
Completed N=27
OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic Fibrosis
Exocrine Pancreatic Insufficiency (EPI) · Cystic Fibrosis (CF)
Source: ClinicalTrials.gov NCT04375878 ↗
Enrolled (actual)
27
Serious AEs
0.0%
Results posted
Sep 2022
Primary outcomePrimary: Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events — 1; 0; 2; 0 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events |
1; 0; 2; 0; 2; 2 | — |
| PRIMARY Efficacy of MS1819: Coefficient of Fat Absorption (CFA) |
66; 83.3; 52.8; 89.3; 52.9; 50.6 | — |
| SECONDARY Stool Weights |
1086.9; 664.4; 1689.6; 644.7; 1301.6; 1409.3 | — |
| SECONDARY Coefficient of Nitrogen Absorption (CNA) |
94.4; 97.3; 90.9; 97.5; 93.1; 92.6 | — |
Eligibility Criteria
Inclusion Criteria
- 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.
- Under stable dose of porcine PERT
- A fair or better nutritional status
- Fecal elastase <100 µg/g
- Standard-of-care medications including 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
Data sourced from ClinicalTrials.gov (NCT04375878). 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. Informational only — not medical advice.