Exocrine Pancreatic Insufficiency (EPI)Phase 2OPTIONNCT03746483
OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
A Phase 2 study testing MS1819 and Porcine PERT in people with Exocrine Pancreatic Insufficiency (EPI) and Cystic Fibrosis (CF), run by Entero Therapeutics at 14 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
- What is being tested?
- MS1819 and Porcine PERT
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 10 Jan 2019to 27 Jul 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
14 sites in 2 countries.
14 sites in 2 countries
United States11 sites
Investigator Site 102
Altamonte Springs, Florida, 32701
Investigator Site 110
Cleveland, Ohio, 44106
Investigator Site 109
Dallas, Texas, 75235
Investigator Site 101
Glenview, Illinois, 60025
Investigator Site 106
Hershey, Pennsylvania, 17033
Investigator Site 103
Las Vegas, Nevada, 89109
Investigator Site 105
Long Beach, California, 90806
Investigator Site 107
Miami, Florida, 33136
Investigator Site 108
Portland, Maine, 04102
Investigator Site 104
Toledo, Ohio, 43606
Investigator Site 111
Wichita, Kansas, 67214
Poland3 sites
Investigator Site 203
Karpacz
Investigator Site 202
Rabka-Zdrój
Investigator Site 204
Sopot
About this study
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
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).
Read the 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.
- Being tested
- MS1819, Porcine PERT
- Keywords
- Exocrine pancreatic insufficiency, EPI, Cystic Fibrosis, CF, Pancreatic enzyme replacement therapy (PERT), Lipase
- Medical subject headings
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MS1819Drug
MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
Porcine PERTDrug
Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention
| Study group | Role | What they receive |
|---|---|---|
| MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks) | Experimental | 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. Receives: Drug: MS1819; Drug: Porcine PERT |
| PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks) | Experimental | 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. Receives: Drug: MS1819; Drug: Porcine PERT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)
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.
Measured: 3 weeks
Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)
Number of participants reporting 1 or more adverse events
Measured: 6 weeks
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
Number of Treatment emergent adverse events
Measured: 6 weeks
Secondary outcomes (3)
Stool Weights
The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights
Measured: 6 weeks
Signs and Symptoms of Malabsorption
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.
Measured: 3 weeks
Coefficient of Nitrogen Absorption (CNA)
CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.
Measured: 3 weeks per group.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2018
- Study start10 Jan 2019
- Primary completion13 Jul 2019
- Study completion27 Jul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Exocrine Pancreatic Insufficiency (EPI)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- 2x2 Crossover
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Entero Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03746483
- Sponsor's protocol ID
- AZ-CF2001
- Acronym
- OPTION
- Status last verified
- Feb 2020
- First submitted
- 15 Nov 2018
- Last updated
- 6 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03746483
- Acronym / sponsor ID
- OPTION / AZ-CF2001
- Keywords
- Exocrine pancreatic insufficiency, EPI, Cystic Fibrosis, CF, Pancreatic enzyme replacement therapy (PERT), Lipase
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Entero TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 10 Jan 2019 (actual)
- Primary completion
- 13 Jul 2019 (actual)
- Study completion
- 27 Jul 2019 (actual)
- First posted
- 19 Nov 2018 (actual)
- Results
- Posted 6 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 2x2 Crossover
| Arm | Type | Receives |
|---|---|---|
| MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks) | Experimental | Drug: MS1819, Drug: Porcine PERTPatients 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. |
| PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks) | Experimental | Drug: MS1819, Drug: Porcine PERTPatients 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MS1819MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement. | Drug | MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks), PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks) |
| Porcine PERTPorcine PERT is being used as a comparator to MS1819 as a second drug/intervention | Drug | MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks), PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks) |
Primary outcome
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.
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
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
Time frame: 6 weeks
Number of Treatment emergent adverse events
3 secondary outcomes
Stool Weights
Time frame: 6 weeks
Signs and Symptoms of Malabsorption
Time frame: 3 weeks
Coefficient of Nitrogen Absorption (CNA)
Time frame: 3 weeks per group.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MS1819 and Porcine PERT in people with Exocrine Pancreatic Insufficiency (EPI) and Cystic Fibrosis (CF), run by Entero Therapeutics at 14 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA) (time frame: 3 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jun 2022.