Exocrine Pancreatic Insufficiency (EPI)Phase 2NCT04375878
OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With 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 15 sites in 2 countries.
Trial 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). 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
- 27 participants
- Where
- 15 sitesin 2 countries
- Study dates
- 20 Jul 2020to 8 Apr 2021
- 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 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
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
15 sites in 2 countries.
15 sites in 2 countries
United States10 sites
Investigator Site 102
Altamonte Springs, Florida, 32701
Investigator Site 110
Cleveland, Ohio, 44106
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
Poland5 sites
Investigator Site 205
Bialystok
Investigator Site 203
Karpacz
Investigator Site 206
Katowice
Investigator Site 202
Rabka-Zdrój
Investigator Site 204
Sopot
About this study
Official title: OPTION 2: A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis; With an Extension Phase Evaluation of Immediate Release MS1819 Capsules
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.
Read the detailed description
This is a Phase 2, open-label, multicenter, 2, 2x2 crossover study assessing the safety and efficacy of MS1819, 2240 mg/day vs porcine PERT, and 4480 mg/day vs porcine PERT given at the same dose and dosing regimen that was being administered during the pre-study period. MS1819 will be administered in enteric capsules.
MS1819 will be assessed in a 2x2 crossover including approximately 30 patients completing both periods. Fifteen patients will be randomized to the MS1819 2240 mg/day vs PERT arm, and 15 patients will be randomized to the MS1819 4480 mg/day vs PERT arm. Patients in each arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.The coefficient of fat absorption (CFA) will be measured at the end of each 3 week study period. Patients enrolled into the extension phase (EP) will be composed of patients who have completed the crossover phase of OPTION 2.
- Being tested
- MS1819, Porcine PERT
- Keywords
- Exocrine Pancreatic Insufficiency, EPI, Cystic Fibrosis, CF
- 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 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement.
Porcine PERTDrug
Porcine PERT is being used a comparator to MS1819 as a second drug/intervention.
| Study group | Role | What they receive |
|---|---|---|
| MS1819 2240 mg/day vs PERT arm, | Active comparator | Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks. Receives: Drug: MS1819; Drug: Porcine PERT |
| MS1819 4480 mg/day vs PERT arm | Active comparator | Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks. Receives: Drug: MS1819; Drug: Porcine PERT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events
Number of subjects reporting 1 or more adverse events
Measured: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Efficacy of MS1819: Coefficient of Fat Absorption (CFA)
The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods.
Measured: 6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase.
Secondary outcomes (2)
Stool Weights
The relative efficacy of MS1819 compared to porcine PERT will be assessed using stool weights.
Measured: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Coefficient of Nitrogen Absorption (CNA)
CNA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. CNAs for MS1819 will be compared to the CNAs of PERT using descriptive methods.
Measured: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2020
- Study start20 Jul 2020
- Primary completion8 Apr 2021
- Study completion8 Apr 2021
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
- 2, 2x2 Crossover
- Masking
- None (open label)
- Enrolment
- 27 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
- NCT04375878
- Sponsor's protocol ID
- AZ-CF2002
- Status last verified
- Aug 2021
- First submitted
- 30 Apr 2020
- Last updated
- 13 Sep 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 13 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- OPTION 2: A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis; With an Extension Phase Evaluation of Immediate Release MS1819 Capsules
- ClinicalTrials.gov ID
- NCT04375878
- Acronym / sponsor ID
- AZ-CF2002
- Keywords
- Exocrine Pancreatic Insufficiency, EPI, Cystic Fibrosis, CF
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Entero TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 20 Jul 2020 (actual)
- Primary completion
- 8 Apr 2021 (actual)
- Study completion
- 8 Apr 2021 (actual)
- First posted
- 6 May 2020 (actual)
- Results
- Posted 13 Sep 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 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.
Detailed description
This is a Phase 2, open-label, multicenter, 2, 2x2 crossover study assessing the safety and efficacy of MS1819, 2240 mg/day vs porcine PERT, and 4480 mg/day vs porcine PERT given at the same dose and dosing regimen that was being administered during the pre-study period. MS1819 will be administered in enteric capsules.
MS1819 will be assessed in a 2x2 crossover including approximately 30 patients completing both periods. Fifteen patients will be randomized to the MS1819 2240 mg/day vs PERT arm, and 15 patients will be randomized to the MS1819 4480 mg/day vs PERT arm. Patients in each arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.The coefficient of fat absorption (CFA) will be measured at the end of each 3 week study period. Patients enrolled into the extension phase (EP) will be composed of patients who have completed the crossover phase of OPTION 2.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 2, 2x2 Crossover
| Arm | Type | Receives |
|---|---|---|
| MS1819 2240 mg/day vs PERT arm, | Active comparator | Drug: MS1819, Drug: Porcine PERTPatients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks. |
| MS1819 4480 mg/day vs PERT arm | Active comparator | Drug: MS1819, Drug: Porcine PERTPatients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks. |
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 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
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 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement. | Drug | MS1819 2240 mg/day vs PERT arm,, MS1819 4480 mg/day vs PERT arm |
| Porcine PERTPorcine PERT is being used a comparator to MS1819 as a second drug/intervention. | Drug | MS1819 2240 mg/day vs PERT arm,, MS1819 4480 mg/day vs PERT arm |
Primary outcome
Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events
Time frame: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Number of subjects reporting 1 or more adverse events
Efficacy of MS1819: Coefficient of Fat Absorption (CFA)
Time frame: 6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase.
The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods.
2 secondary outcomes
Stool Weights
Time frame: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Coefficient of Nitrogen Absorption (CNA)
Time frame: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 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.
15 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 15 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 13 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 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 13 Sep 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?
Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events (time frame: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)). The study lists 2 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 13 Sep 2022.