Short Bowel SyndromePhase 2NCT04046328
Efficacy, Safety and Tolerability of Enteric-Coated Cholestyramine Capsules for Adult Short Bowel Syndrome
A Phase 2 study testing Enteric-Coated Cholestyramine (ECC) Capsule in people with Short Bowel Syndrome, run by Pharmascience Inc. at 3 sites in Poland.
Trial summary
A new Enteric-Coated Cholestyramine (ECC) capsule has been developed to manage diarrhea associated with Short Bowel Syndrome (SBS) in adults. The formulation is expected to release cholestyramine in the remaining segment of the small intestine in SBS patients, thus binding bile acids after fat digestion, but before… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Short Bowel Syndrome
- What is being tested?
- Enteric-Coated Cholestyramine (ECC) Capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 13 participants
- Where
- 3 sitesPoland
- Study dates
- 15 Oct 2019to 22 Dec 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult, ambulatory male and female subjects
- Provision of signed and dated informed consent form (ICF)
- Age ≥ 18 years and ≤ 80 years
- Stable SBS of:
- Non-surgical origin; OR
- Surgical origin where the last surgical ileal resection was performed at least 6 months prior to enrolment
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- Partial, Home Parenteral Nutrition and/or parenteral fluids are allowed, at a maximum frequency of 6 times a week throughout the trial, as long as the regimen has been stable for at least 2 weeks prior to screening and is expected to remain unchanged during the study
- At least 50 % of the colon being intact
- Intact duodenum
- BMI ≥ 18
- Presence of stable chronic diarrhea for at least 3 months prior to enrolment as evidenced by medical history
- Presence of stable chronic diarrhea during the 2-week screening diary period before randomization, as evidenced by completion of a screening diary demonstrating:
- Mean daily production of at least 3 soft or watery stools (BSFS scores 6 or 7); or
- More than 3 bowel movements per day on average with >25% of them being BSFS type 6 or 7
- Stated willingness and ability to comply with all study procedures, including daily recording of bowel movements and BSFS in the patient diaries, and availability for the duration of the study
- Clinical laboratory values within the laboratory's stated normal range; if not within this range, they must be without clinical significance, as determined by the investigator
- Female subjects must meet one of the following criteria:
- Participant is of childbearing potential and agrees to use one of the accepted contraceptive regimens from at least 30 days prior to the first study treatment administration through to at least 30 days after the last dose of the study treatment. An acceptable method of contraception includes one of the following:
- Abstinence from heterosexual intercourse
- Systemic contraceptives (combined birth control pills, injectable/implant/insertable hormonal birth control products, transdermal patch)
- Intrauterine device (with or without hormones)
- Condom with spermicide
- Participants of non-childbearing potential, defined as surgically sterile (i.e. has undergone complete hysterectomy or bilateral oophorectomy) or is in a menopausal state (i.e. at least 1 year without menses prior to the first study drug administration) are eligible
Who cannot join
- Patients with known or suspected intestinal strictures of clinical relevance as judged by the Investigator
- Active inflammatory bowel disease (IBD) or fistula during the screening period as judged by the Investigator
- Crohn's disease patients not being in clinical remission for the last 12 weeks prior to randomization
- Diarrhea caused by other causes than SBS
- Presence of clinically significant steatorrhea, requiring pancreatic enzymes supplementation
- Presence of complete biliary obstruction
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- Presence of active cancer (except resected cutaneous basal or squamous cell carcinoma and except in situ cervical cancer) and/or need to receive chemotherapy or radiotherapy during the study
- History of allergic reaction to cholestyramine or any excipient of the investigational drug product or placebo, or packaging components
- Females who are lactating at screening
- Females who are pregnant according to the pregnancy test at screening or prior to the first study treatment administration
- Significant history (at least 3 consecutive months in the year prior to Screening) of drug dependency or alcohol abuse (> 3 units of alcohol per day, intake of excessive alcohol, acute or chronic)
- Subjects who took an Investigational Product (IP) in the 30 days prior to the first study drug administration
- Any other clinically significant condition that is considered by the principal investigator as being susceptible to put the patient at greater safety risk, influence response to study product, or interfere with study assessments.
Trial locations and contacts
3 sites in 1 country.
Poland3 sites
Wojewódzki Specjalistyczny Szpital im. M. Pirogowa w Łodzi
Lodz
Solumed Centrum Medyczne
Poznan
Szpital Wielospecjalistyczny im. Stanleya Dudricka
Skawina
About this study
Official title: A Phase IIa, Proof of Concept, Randomized, Double-Blind, Dose-Finding, Cross-Over Study of the Efficacy, Safety and Tolerability of a New Enteric-Coated Cholestyramine Capsule in Adult Short Bowel Syndrome Patients
A new Enteric-Coated Cholestyramine (ECC) capsule has been developed to manage diarrhea associated with Short Bowel Syndrome (SBS) in adults. The formulation is expected to release cholestyramine in the remaining segment of the small intestine in SBS patients, thus binding bile acids after fat digestion, but before induction of diarrhea in the colon. The delayed-release profile is also expected to help reduce the potential for drug-drug interactions occurring in the proximal small intestine. Two doses of ECC will be studied for efficacy, safety and tolerability in this Phase IIa trial.
Read the detailed description
A new Enteric-Coated Cholestyramine (ECC) capsule has been developed to manage diarrhea associated with Short Bowel Syndrome (SBS). SBS is usually caused by the significant resection or loss of function of the ileum, leading to reduced reabsorption of bile acids and subsequent osmotic diarrhea. The new ECC formulation could release cholestyramine in the remaining segment of the small intestine in SBS patients, delivering and binding bile acids before they induce diarrhea in the colon. The proposed advantages of this formulation are: a) to prevent drug-drug interactions in the proximal GI tract, b) to preserve the fat digestive properties of bile acids in the duodenum and 3) to offer a more palatable dosage form to patients. Moreover, since distal delivery of cholestyramine is expected to be more effective in diarrhea prevention/reduction in SBS, lower doses than the ones used with non-enteric coated cholestyramine may be sufficient. Two doses of ECC will be studied for efficacy, safety and tolerability in well-defined non fully-colectomized, adult SBS patients suffering from diarrhea.
- Conditions
- Short Bowel Syndrome
- Being tested
- Enteric-Coated Cholestyramine (ECC) Capsule, Placebo
- Medical subject headings
- Short Bowel Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Enteric-Coated Cholestyramine (ECC) CapsuleDrug
Enteric-Coated Delayed Release Cholestyramine Capsules
PlaceboDrug
Enteric-Coated Delayed Release Placebo Capsules
| Study group | Role | What they receive |
|---|---|---|
| "Low" Dose ECC Regimen | Experimental | ECC at the 1.7 g daily dose, administered BID (twice daily) as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal. Receives: Drug: Enteric-Coated Cholestyramine (ECC) Capsule; Drug: Placebo |
| "High" Dose ECC Regimen | Experimental | ECC at the 4.25 g daily dose, administered BID (twice daily) as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal. Receives: Drug: Enteric-Coated Cholestyramine (ECC) Capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the Weekly Frequency of Bowel Movements Measured Between Baseline and the Second Week of Treatment
Change in the weekly frequency of bowel movements measured between baseline and the second week of treatment. Baseline is defined as the second week of screening for treatment period 1 and second week of washout for treatment period 2.
Measured: Baseline and Week 2 of treatment (Days 8 to 14, and Days 36 to 42)
Secondary outcomes (3)
Total Number of Bowel Movements for the Whole 2-week Treatment Period
Measured: Days 1 to 14 and Days 29 to 42
Mean Daily Stool Form Score According to the BSFS (Bristol Stool Form Scale), Measured During the Second Week of Treatment
The BSFS classifies the form of human feces into seven categories (Type 1 to Type 7) based on stool shape and consistency. Types or scores of 1 and 2 indicate constipation, with 3 and 4 being the ideal stools as they are easy to defecate while not containing excess liquid, 5 tending towards diarrhea, and 6 and 7 indicate diarrhea.
Measured: Days 8 to 14, and Days 36 to 42
Mean Daily Dose of Loperamide in mg, if Used, During the Second Week of Treatment
Measured: Days 8 to 14, and Days 36 to 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2019
- Study start15 Oct 2019
- Primary completion22 Dec 2021
- Study completion22 Dec 2021
Why it stopped: Difficult recruitment
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 Short Bowel SyndromeStudies 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
- Multiple-center, randomized, double-blind, double dummy, 2-period, 2-sequence cross-over design
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharmascience Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Marek Kunecki, MD, PhD, Principal investigator, Wojewódzki Specjalistyczny Szpital im. M. Pirogowa w ŁodziKonrad Matysiak, MD, PhD, Principal investigator, Solumed Centrum MedyczneKinga Szczepanek, MD, Principal investigator, Szpital Wielospecjalistyczny im. Stanleya Dudricka
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04046328
- Sponsor's protocol ID
- PMS-2018-002
- Other IDs
- 2018-004419-32, 2018
- Status last verified
- Jun 2022
- First submitted
- 26 Jul 2019
- Last updated
- 19 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- 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 19 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa, Proof of Concept, Randomized, Double-Blind, Dose-Finding, Cross-Over Study of the Efficacy, Safety and Tolerability of a New Enteric-Coated Cholestyramine Capsule in Adult Short Bowel Syndrome Patients
- ClinicalTrials.gov ID
- NCT04046328
- Acronym / sponsor ID
- PMS-2018-002
- Conditions
- Short Bowel Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Difficult recruitment
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Pharmascience Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Oct 2019 (actual)
- Primary completion
- 22 Dec 2021 (actual)
- Study completion
- 22 Dec 2021 (actual)
- First posted
- 6 Aug 2019 (actual)
- Results
- Posted 19 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A new Enteric-Coated Cholestyramine (ECC) capsule has been developed to manage diarrhea associated with Short Bowel Syndrome (SBS) in adults. The formulation is expected to release cholestyramine in the remaining segment of the small intestine in SBS patients, thus binding bile acids after fat digestion, but before induction of diarrhea in the colon. The delayed-release profile is also expected to help reduce the potential for drug-drug interactions occurring in the proximal small intestine. Two doses of ECC will be studied for efficacy, safety and tolerability in this Phase IIa trial.
Detailed description
A new Enteric-Coated Cholestyramine (ECC) capsule has been developed to manage diarrhea associated with Short Bowel Syndrome (SBS). SBS is usually caused by the significant resection or loss of function of the ileum, leading to reduced reabsorption of bile acids and subsequent osmotic diarrhea. The new ECC formulation could release cholestyramine in the remaining segment of the small intestine in SBS patients, delivering and binding bile acids before they induce diarrhea in the colon. The proposed advantages of this formulation are: a) to prevent drug-drug interactions in the proximal GI tract, b) to preserve the fat digestive properties of bile acids in the duodenum and 3) to offer a more palatable dosage form to patients. Moreover, since distal delivery of cholestyramine is expected to be more effective in diarrhea prevention/reduction in SBS, lower doses than the ones used with non-enteric coated cholestyramine may be sufficient. Two doses of ECC will be studied for efficacy, safety and tolerability in well-defined non fully-colectomized, adult SBS patients suffering from diarrhea.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Multiple-center, randomized, double-blind, double dummy, 2-period, 2-sequence cross-over design
| Arm | Type | Receives |
|---|---|---|
| "Low" Dose ECC Regimen | Experimental | Drug: Enteric-Coated Cholestyramine (ECC) Capsule, Drug: PlaceboECC at the 1.7 g daily dose, administered BID (twice daily) as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal. |
| "High" Dose ECC Regimen | Experimental | Drug: Enteric-Coated Cholestyramine (ECC) CapsuleECC at the 4.25 g daily dose, administered BID (twice daily) as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Adult, ambulatory male and female subjects
- Provision of signed and dated informed consent form (ICF)
- Age ≥ 18 years and ≤ 80 years
- Stable SBS of:
- Partial, Home Parenteral Nutrition and/or parenteral fluids are allowed, at a maximum frequency of 6 times a week throughout the trial, as long as the regimen has been stable for at least 2 weeks prior to screening and is expected to remain unchanged during the study
Key exclusion criteria (13 listed)
- Patients with known or suspected intestinal strictures of clinical relevance as judged by the Investigator
- Active inflammatory bowel disease (IBD) or fistula during the screening period as judged by the Investigator
- Crohn's disease patients not being in clinical remission for the last 12 weeks prior to randomization
- Diarrhea caused by other causes than SBS
- Presence of clinically significant steatorrhea, requiring pancreatic enzymes supplementation
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 |
|---|---|---|
| Enteric-Coated Cholestyramine (ECC) CapsuleEnteric-Coated Delayed Release Cholestyramine Capsules | Drug | "High" Dose ECC Regimen, "Low" Dose ECC Regimen |
| PlaceboEnteric-Coated Delayed Release Placebo Capsules | Drug | "Low" Dose ECC Regimen |
Primary outcome
Change in the Weekly Frequency of Bowel Movements Measured Between Baseline and the Second Week of Treatment
Time frame: Baseline and Week 2 of treatment (Days 8 to 14, and Days 36 to 42)
Change in the weekly frequency of bowel movements measured between baseline and the second week of treatment. Baseline is defined as the second week of screening for treatment period 1 and second week of washout for treatment period 2.
3 secondary outcomes
Total Number of Bowel Movements for the Whole 2-week Treatment Period
Time frame: Days 1 to 14 and Days 29 to 42
Mean Daily Stool Form Score According to the BSFS (Bristol Stool Form Scale), Measured During the Second Week of Treatment
Time frame: Days 8 to 14, and Days 36 to 42
Mean Daily Dose of Loperamide in mg, if Used, During the Second Week of Treatment
Time frame: Days 8 to 14, and Days 36 to 42
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 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.
Marek Kunecki, MD, PhD principal investigator
Wojewódzki Specjalistyczny Szpital im. M. Pirogowa w Łodzi
Konrad Matysiak, MD, PhD principal investigator
Solumed Centrum Medyczne
Kinga Szczepanek, MD principal investigator
Szpital Wielospecjalistyczny im. Stanleya Dudricka
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 Enteric-Coated Cholestyramine (ECC) Capsule in people with Short Bowel Syndrome, run by Pharmascience Inc. at 3 sites in Poland.
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 23 inclusion and 13 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: stopped early, as of the sponsor's last update on 19 Jul 2022. 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 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 19 Jul 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?
Change in the Weekly Frequency of Bowel Movements Measured Between Baseline and the Second Week of Treatment (time frame: Baseline and Week 2 of treatment (Days 8 to 14, and Days 36 to 42)). 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 Jul 2022.