Exocrine Pancreatic InsufficiencyPhase 2SPANNCT05719311
Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis
A Phase 2 study testing adrulipase in people with Exocrine Pancreatic Insufficiency and Cystic Fibrosis, run by Entero Therapeutics at 3 sites in United States.
Trial summary
Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI). Currently, these patients take pancreatic enzymes that are obtained from pig pancreas to aid the digestion of food. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- What is being tested?
- adrulipase
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Feb 2023to 28 Jul 2023
- 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
- A confirmed diagnosis of cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride >60 mmol/L by quantitative pilocarpine iontophoresis (measured while not on a CFTR modulator) or genotype.
- On stable dose of porcine PERT ≥1 month (30 days) prior to screening; stable dose is defined as dose of medication not changed during this time period, and the medication must be commercially available and be administered in the recommended dose range.
- CFA = or > 80% at screening while on stable PERT
- A fair or better nutritional status as defined by:
- BMI ≥16.0 kg/m2 for female patients ≥18 years of age, or
- BMI ≥16.5 kg/m2 for male patients ≥18 years of age
- Fecal elastase <100 µg/g of stool at screening
- 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 Screening
- Feeding via an enteral tube during 6 months before screening
- Forced expiratory volume ≤30% at the Screening visit
- Changes in gastric acid suppressant therapy during the one month prior to screening for patients already on suppressant therapy.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Childrens Lung Specialists
Las Vegas, Nevada, 89109
The Cystic Fibrosis Institute
Northfield, Illinois, 60093
Central Florida Pulmonary Group
Orlando, Florida, 32803
About this study
Official title: SPAN: A Phase 2, Open Label, Multicenter, Pilot Study to Assess Safety and Efficacy of an Enteric Microgranule Formulation of Adrulipase in Patients With Exocrine Pancreatic Insufficiency (EPI) Due to Cystic Fibrosis (CF)
Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI). Currently, these patients take pancreatic enzymes that are obtained from pig pancreas to aid the digestion of food. The goals of this clinical study are to evaluate the safety and efficacy of a novel formulation of a non-porcine lipase, called adrulipase, in patients with EPI due to cystic fibrosis.
The main question[s] the study aims to answer are:
Read more
- Is the novel formulation of adrulipase safe to use at the doses being evaluated in the clinical study.
- Is adrulipase as effective, or more effective, compared to the pig enzymes the patients currently use.
Researchers will compare the results obtained with adrulipase to how the patients typically respond to their pig enzymes to see if adrulipase helps patients digest fats adequately and if their stomach feels good (signs and symptoms of malabsorption).
Read the detailed description
This is an Phase 2, open label, single arm pilot study assessing the safety and efficacy of adrulipase in an enteric microgranule formulation. Patients with a confirmed diagnosis of cystic fibrosis who are 18 years of age or greater will be screened for eligibility if they have been clinically controlled on a stable dose of commercial pancreatic enzyme replacement therapy (PERT) for at least one month. Patients on cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies must have been on a stable dose for at least 3 months prior to study entry, and no dose changes will be made during the study. Patients receiving gastric acid suppressants must have been on a stable dose for at least one month prior to study entry and no dose changes will be made during the study.
Upon obtaining an informed consent, potentially eligible patients will receive dietary counselling during the week prior to the scheduled date of confinement for collecting stool samples for calculation of baseline coefficient of fat absorption (CFA). This counselling will emphasize the importance of dietary stability during the study. Patients found to have a CFA of 80% or greater while receiving their commercial PERT and meeting the other eligibility criteria will be enrolled into the study.
Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. The patient will remain on study for approximately three weeks, after which a repeat CFA will be obtained. A dose titration scheme will be used for determining whether a low, medium, or high dose of adrulipase may succeed in controlling signs and symptoms of exocrine pancreatic insufficiency (EPI) and provide a CFA of 80% or greater. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase. After patients reach 3 weeks of study and complete their end of study CFA, they will be returned to their pre-study commercial PERT. An end of study safety visit will be scheduled for one week after finishing adrulipase therapy.
Safety assessments will be made by collecting adverse events, safety lab assessments, and immunologic assays to assess drug induced immune responses.
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Being tested
- adrulipase
- 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.
adrulipaseDrug
Also called MS1819
Enteric microgranule formulation of adrulipase.
| Study group | Role | What they receive |
|---|---|---|
| Adrulipase | Experimental | Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase. Receives: Drug: adrulipase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of Adrulipase
Number of subjects reporting 1 or more adverse events.
Measured: End of 3-week treatment period.
Efficacy of Adrulipase: Coefficient of Fat Absorption (CFA)
The primary efficacy endpoint is the CFA that will be assessed at the end of the 3-week treatment period. CFAs for adrulipase will be compared to the CFAs of PERT obtained at baseline/eligibility using descriptive methods.
Measured: End of 3-week treatment period.
Secondary outcomes (2)
Stool Weight
Stool weights obtained during the supervised confinement visit at the end of the 3-week treatment period will be measured. Stool weights obtained during confinement on adrulipase will be compared to the stool weights during confinement on PERT obtained at baseline/eligibility using descriptive methods.
Measured: Change between two time points: initial PERT confinement and end of 3-week treatment period.
Coefficient of Nitrogen Absorption (CNA)
CNA that will be assessed at the end of the 3-week treatment period. CNAs for adrulipase will be compared to the CNAs of PERT obtained at baseline/eligibility using descriptive methods.
Measured: Change between two time points: initial PERT confinement and end of 3-week treatment period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2023
- Study start1 Feb 2023
- Primary completion20 Jun 2023
- Study completion28 Jul 2023
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 InsufficiencyStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 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
- NCT05719311
- Sponsor's protocol ID
- FWB-CF-2.03
- Acronym
- SPAN
- Status last verified
- Jul 2023
- First submitted
- 31 Jan 2023
- Last updated
- 19 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SPAN: A Phase 2, Open Label, Multicenter, Pilot Study to Assess Safety and Efficacy of an Enteric Microgranule Formulation of Adrulipase in Patients With Exocrine Pancreatic Insufficiency (EPI) Due to Cystic Fibrosis (CF)
- ClinicalTrials.gov ID
- NCT05719311
- Acronym / sponsor ID
- SPAN / FWB-CF-2.03
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Entero TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Feb 2023 (actual)
- Primary completion
- 20 Jun 2023 (actual)
- Study completion
- 28 Jul 2023 (actual)
- First posted
- 8 Feb 2023 (actual)
- Results
- Posted 19 Sep 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI). Currently, these patients take pancreatic enzymes that are obtained from pig pancreas to aid the digestion of food. The goals of this clinical study are to evaluate the safety and efficacy of a novel formulation of a non-porcine lipase, called adrulipase, in patients with EPI due to cystic fibrosis.
The main question[s] the study aims to answer are:
- Is the novel formulation of adrulipase safe to use at the doses being evaluated in the clinical study.
- Is adrulipase as effective, or more effective, compared to the pig enzymes the patients currently use.
Researchers will compare the results obtained with adrulipase to how the patients typically respond to their pig enzymes to see if adrulipase helps patients digest fats adequately and if their stomach feels good (signs and symptoms of malabsorption).
Detailed description
This is an Phase 2, open label, single arm pilot study assessing the safety and efficacy of adrulipase in an enteric microgranule formulation. Patients with a confirmed diagnosis of cystic fibrosis who are 18 years of age or greater will be screened for eligibility if they have been clinically controlled on a stable dose of commercial pancreatic enzyme replacement therapy (PERT) for at least one month. Patients on cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies must have been on a stable dose for at least 3 months prior to study entry, and no dose changes will be made during the study. Patients receiving gastric acid suppressants must have been on a stable dose for at least one month prior to study entry and no dose changes will be made during the study.
Upon obtaining an informed consent, potentially eligible patients will receive dietary counselling during the week prior to the scheduled date of confinement for collecting stool samples for calculation of baseline coefficient of fat absorption (CFA). This counselling will emphasize the importance of dietary stability during the study. Patients found to have a CFA of 80% or greater while receiving their commercial PERT and meeting the other eligibility criteria will be enrolled into the study.
Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. The patient will remain on study for approximately three weeks, after which a repeat CFA will be obtained. A dose titration scheme will be used for determining whether a low, medium, or high dose of adrulipase may succeed in controlling signs and symptoms of exocrine pancreatic insufficiency (EPI) and provide a CFA of 80% or greater. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase. After patients reach 3 weeks of study and complete their end of study CFA, they will be returned to their pre-study commercial PERT. An end of study safety visit will be scheduled for one week after finishing adrulipase therapy.
Safety assessments will be made by collecting adverse events, safety lab assessments, and immunologic assays to assess drug induced immune responses.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adrulipase | Experimental | Drug: adrulipaseUpon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- A confirmed diagnosis of cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride >60 mmol/L by quantitative pilocarpine iontophoresis (measured while not on a CFTR modulator) or genotype.
- On stable dose of porcine PERT ≥1 month (30 days) prior to screening; stable dose is defined as dose of medication not changed during this time period, and the medication must be commercially available and be administered in the recommended dose range.
- CFA = or > 80% at screening while on stable PERT
- A fair or better nutritional status as defined by:
- Fecal elastase <100 µg/g of stool at screening
Key exclusion criteria (7 listed)
- History or diagnosis of fibrosing colonopathy
- Any chronic diarrheal illness unrelated to pancreatic insufficiency
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level
- 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 |
|---|---|---|
| adrulipaseAlso: MS1819Enteric microgranule formulation of adrulipase. | Drug | Adrulipase |
Primary outcome
Safety of Adrulipase
Time frame: End of 3-week treatment period.
Number of subjects reporting 1 or more adverse events.
Efficacy of Adrulipase: Coefficient of Fat Absorption (CFA)
Time frame: End of 3-week treatment period.
The primary efficacy endpoint is the CFA that will be assessed at the end of the 3-week treatment period. CFAs for adrulipase will be compared to the CFAs of PERT obtained at baseline/eligibility using descriptive methods.
2 secondary outcomes
Stool Weight
Time frame: Change between two time points: initial PERT confinement and end of 3-week treatment period.
Coefficient of Nitrogen Absorption (CNA)
Time frame: Change between two time points: initial PERT confinement and end of 3-week treatment period
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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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.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing adrulipase in people with Exocrine Pancreatic Insufficiency and Cystic Fibrosis, run by Entero Therapeutics at 3 sites in United States.
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 8 inclusion and 7 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 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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 19 Sep 2024). 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 Adrulipase (time frame: End of 3-week treatment 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 19 Sep 2024.