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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-01-10

Study Completion Date

2019-07-27

Brief Summary

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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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Detailed Description

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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.

Conditions

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Exocrine Pancreatic Insufficiency (EPI) Cystic Fibrosis (CF)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

2x2 Crossover

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Unblinded

Study Groups

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MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks)

Patients in arm will further be randomized to receive either the sequence consisting of MS1819 2240 mg/day for 3 weeks followed by PERT for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.

Group Type EXPERIMENTAL

MS1819

Intervention Type DRUG

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

Porcine PERT

Intervention Type DRUG

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

PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)

Patients in arm will be randomized to receive PERT for 3 weeks followed by MS1819 2240 mg/day for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.

Group Type EXPERIMENTAL

MS1819

Intervention Type DRUG

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

Porcine PERT

Intervention Type DRUG

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

Interventions

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MS1819

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

Intervention Type DRUG

Porcine PERT

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

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No