Short Bowel SyndromePhase 3NCT02242656
A Phase III Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Investigational Product MP-101 in Subjects With Short Bowel Syndrome Who Have Had an Inadequate Response to Anti-Diarrheals
A Phase 3 study testing Opium Tincture USP Deodorized in people with Short Bowel Syndrome and Short Gut Syndrome, run by Marathon Pharmaceuticals, LLC at 5 sites in United States.
Trial summary
MP-101 will be evaluated in this study to see if it is safe, tolerable, and can help people with Short Bowel Syndrome. This study will also find out if taking MP-101 can improve the symptoms of Short Bowel Syndrome and reduce the number of times subjects experience bowel movements. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Short Bowel Syndrome, Short Gut Syndrome, SBS
- What is being tested?
- Opium Tincture USP Deodorized
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 5 sitesUnited States
- Study dates
- Dec 2014to Dec 2017
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Be male or female adults, 18 years of age or older at the time of consent
- Have SBS that is inadequately controlled on current antidiarrheal medication (e.g., loperamide or diphenoxylate), including subjects with ileostomies, based on the 7 days prior to Day 1 of the study
- Subjects must be >3months post intestinal resection
- Have a history of persistent loose stools for more than 4 weeks
- Be on a combination of Opium Tincture and an anti-diarrheal (loperamide or diphenoxylate) or an anti-diarrheal agent alone
- If currently taking Opium Tincture, be willing to stop the continued use of Opium Tincture at screening visit until the start of study treatment and willing to stop the use of any other anti-diarrheal for the duration of the study
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- Be able to maintain their current diet for the duration of the study
- Be on stable nutritional support (parenteral or oral)
- Males or non-pregnant, non-lactating females who are postmenopausal, naturally or surgically sterile, or who agree to use effective contraceptive methods throughout the course of the study. Postmenopausal is defined as at least 12 months of natural spontaneous amenorrhea, or at least 6 weeks following surgical menopause (bilateral oophorectomy)
- Females of childbearing potential must agree to use 1 of the following acceptable birth control methods:
- Surgically sterile (hysterectomy or bilateral oophorectomy)
- Surgically sterile (bilateral tubal ligation with surgery at least 6 weeks prior to study initiation)
- Intrauterine device (IUD) in place for at least 3 months
- Abstinence (not having sexual intercourse)
- Barrier method (condom or diaphragm) with spermicide for at least 14 days prior to screening and through study completion
- Stable hormonal contraceptive for at least 3 months prior to study and through study completion
- Vasectomized partner
- Females of childbearing potential must have a negative serum human chorionic gonadotropin (hCG) pregnancy test at screening
- Be able to understand and provide signed informed consent
Who cannot join
- Unable or unwilling to stop the use of Opium Tincture or any anti-diarrheal medication at the screening visit.
- Have any history of or active neurological, endocrine, cardiovascular, pulmonary, hematological, immunologic, psychiatric, or metabolic disease that is considered clinically significant, is not currently controlled by medication, and is stable as deemed by the Investigator
- Have clinically significant electrocardiogram (ECG) abnormalities as determined by the PI or vital sign abnormalities (systolic blood pressure < 90 mmHg, diastolic blood pressure < 60 mmHg, or heart rate >100 bpm) at screening
- Have clinically significant elevation of liver enzymes (> 3 times the upper limit of normal) or clinically relevant renal disease, (creatinine >1.5) or any other clinically significant abnormal laboratory test results found during medical screening as determined by the Principal Investigator
- Have a history of major mental illness that in the opinion of the Investigator may affect the ability of the subject to safely participate and reliably complete the study
- Have a history of alcohol or substance abuse within the past 2 years. Alcohol abuse will be defined as >14 drinks per week (1 drink = 12 oz. beer, 5.0 oz. wine, or 1.5 oz. distilled spirits)
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- Have a known allergy or intolerance to Opium Tincture or any of the excipients in the formulation (alcohol, opium, or morphine)
- Is currently taking an opioid derivative (other than Opium Tincture) or any other medication which, in the opinion of the investigator, could interfere with the interpretation of the study results
- Are currently taking antibiotics for bacterial overgrowth
- Have participated in another interventional clinical trial within 30 days prior to screening with the exception of observational cohort studies or non-interventional studies.
- Have known or suspected pregnancy, planned pregnancy, or lactation
- Have a planned surgery over the course of the study
- Have a condition the Investigator believes would interfere with the ability to provide informed consent or comply with study instructions, or that might confound the interpretation of the study results or put the subject at undue risk
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60611
Cleveland Clinic
Cleveland, Ohio, 44195
Indiana University
Indianapolis, Indiana, 46202
Regional Infectious Diseases Infusion Center
Lima, Ohio, 45801
Vanderbilt Center for Human Nutrition
Nashville, Tennessee, 37212-1150
About this study
Official title: A Phase 3, Open Label Responder Treatment Period With a Randomized, Blinded, Three-Period Crossover, Followed by an Open Label Safety Period to Evaluate the Clinical Efficacy, Safety, and Pharmacokinetics of Investigational Drug MP-101 (Opium Tincture and Opium Tincture With Reduced Uncharacterized Material) to Treat Chronic Diarrhea in Subjects With Short Bowel Syndrome Who Have Had an Inadequate Response to Anti-Diarrheals
MP-101 will be evaluated in this study to see if it is safe, tolerable, and can help people with Short Bowel Syndrome. This study will also find out if taking MP-101 can improve the symptoms of Short Bowel Syndrome and reduce the number of times subjects experience bowel movements.
- Conditions
- Short Bowel Syndrome, Short Gut Syndrome, SBS, Short Bowel, Short Gut
- Being tested
- Opium Tincture USP Deodorized
- Keywords
- Short Bowel Syndrome, SBS, Chronic Diarrhea, Short Bowel, Short Gut, Short Gut Syndrome, Anti-Diarrheal, Opium Tincture, loperamide, diphenoxylate, ileostomies, tincture of opium, DTO, codeine, morphine, opiate
- 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.
Opium Tincture USP DeodorizedDrug
Treatment A - 0.6 mL, MP-101 (10 mg/mL, Opium Tincture (OT)), USP (Deodorized) QID oral drops, followed by Treatment B - 0.6 mL, MP-101, 1/12 dilution, (0.833 mg/mL, OT), USP (Deodorized) QID oral drops, followed by Treatment C - 0.6 mL, MP-101 (10 mg/mL Opium Tincture with reduced uncharacterized material) QID oral drops
Opium Tincture USP DeodorizedDrug
Treatment B - 0.6 mL, MP-101, 1/12 dilution, (0.833 mg/mL, OT), USP (Deodorized) QID oral drops, followed by Treatment A - 0.6 mL, MP-101 (10 mg/mL, Opium Tincture (OT)), USP (Deodorized) QID oral drops, followed by Treatment C - 0.6 mL, MP-101 (10 mg/mL Opium Tincture with reduced uncharacterized material) QID oral drops
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Assigned to receive Treatment A (Period 1), Treatment B (Period 2), Treatment C (Period 3) Receives: Drug: Opium Tincture USP Deodorized |
| Group 2 | Experimental | Assigned to receive Treatment B (Period 1), Treatment A (Period 2), Treatment C (Period 3) Receives: Drug: Opium Tincture USP Deodorized |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of responders between Treatments A and B for the Intent-To-Treat (ITT) population
30% or greater reduction in 24-hour stool volume from baseline
Measured: Last day of Periods 1 (Days 16-17) and 2 (Days 25-26)
Secondary outcomes (8)
Percentage of responders between Treatments A and B for the Modified-Intent-To-Treat (mITT) population
30% or greater reduction in 24-hour stool volume from baseline for subjects who complete both periods 1 and 2 of the randomized, blinded, crossover portion of the study
Measured: Last day of Periods 1 (Days 16-17) and 2 (Days 25-26)
Establish non-inferiority of the percentage of responders between Treatment A and Treatment C
30% or greater reduction in stool volume from baseline between Treatment A (with uncharacterized material) and Treatment C (with reduced uncharacterized material) for ITT and mITT efficacy population (subjects from the ITT population who complete both Treatments A and C)
Measured: Last day of Period 3 (Day 34-35)
24-hour stool volume comparison between Treatment A and B for ITT and mITT
Comparison of stool volume measurements
Measured: Last day of Periods 1 (Day 16-17) and 2 (Day 25-26)
24-hour fecal events between Treatment A and B for ITT and mITT
Changes in the number of 24-hour fecal events for ITT and mITT
Measured: Period 1 (Day 10-16) and 2 (Day 19-25)
24-hour nocturnal fecal events between Treatment A and B for ITT and mITT
Changes in the number of 24-hour nocturnal (ad defined for each subject) fecal events for ITT and mITT
Measured: Period 1 (Day 10-16) and 2 (Day 19-25)
Single and multiple dose Pharmacokinetic (PK) parameters
Evaluate the pharmacokinetics of morphine, codeine, thebaine and papaverine after single dose and multiple dose administration of MP-101
Measured: Day-1, Day 1, Day 7, Day 57-59
Number of Participants with Adverse Events
Review of safety events throughout the course of the study
Measured: Up to 60 Days
Number of Participants Up-Titrated
Review of safety events throughout the course of the study
Measured: Days 35-60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2014 (estimated)
- Study startDec 2014
- Primary completionDec 2017 (estimated)
Why it stopped: Transfer of IND to new marketing authorization holder
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Marathon Pharmaceuticals, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02242656
- Sponsor's protocol ID
- MP-101-CL-001
- Status last verified
- Oct 2014
- First submitted
- 10 Sep 2014
- Last updated
- 18 Feb 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Open Label Responder Treatment Period With a Randomized, Blinded, Three-Period Crossover, Followed by an Open Label Safety Period to Evaluate the Clinical Efficacy, Safety, and Pharmacokinetics of Investigational Drug MP-101 (Opium Tincture and Opium Tincture With Reduced Uncharacterized Material) to Treat Chronic Diarrhea in Subjects With Short Bowel Syndrome Who Have Had an Inadequate Response to Anti-Diarrheals
- ClinicalTrials.gov ID
- NCT02242656
- Acronym / sponsor ID
- MP-101-CL-001
- Conditions
- Short Bowel Syndrome, Short Gut Syndrome, SBS, Short Bowel, Short Gut
- Keywords
- Short Bowel Syndrome, SBS, Chronic Diarrhea, Short Bowel, Short Gut, Short Gut Syndrome, Anti-Diarrheal, Opium Tincture
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Transfer of IND to new marketing authorization holder
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Marathon Pharmaceuticals, LLCIndustry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- Dec 2017 (estimated)
- First posted
- 17 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
MP-101 will be evaluated in this study to see if it is safe, tolerable, and can help people with Short Bowel Syndrome. This study will also find out if taking MP-101 can improve the symptoms of Short Bowel Syndrome and reduce the number of times subjects experience bowel movements.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Drug: Opium Tincture USP DeodorizedAssigned to receive Treatment A (Period 1), Treatment B (Period 2), Treatment C (Period 3) |
| Group 2 | Experimental | Drug: Opium Tincture USP DeodorizedAssigned to receive Treatment B (Period 1), Treatment A (Period 2), Treatment C (Period 3) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Be male or female adults, 18 years of age or older at the time of consent
- Have SBS that is inadequately controlled on current antidiarrheal medication (e.g., loperamide or diphenoxylate), including subjects with ileostomies, based on the 7 days prior to Day 1 of the study
- Have a history of persistent loose stools for more than 4 weeks
- Be on a combination of Opium Tincture and an anti-diarrheal (loperamide or diphenoxylate) or an anti-diarrheal agent alone
- If currently taking Opium Tincture, be willing to stop the continued use of Opium Tincture at screening visit until the start of study treatment and willing to stop the use of any other anti-diarrheal for the duration of the study
Key exclusion criteria (13 listed)
- Unable or unwilling to stop the use of Opium Tincture or any anti-diarrheal medication at the screening visit.
- Have any history of or active neurological, endocrine, cardiovascular, pulmonary, hematological, immunologic, psychiatric, or metabolic disease that is considered clinically significant, is not currently controlled by medication, and is stable as deemed by the Investigator
- Have clinically significant electrocardiogram (ECG) abnormalities as determined by the PI or vital sign abnormalities (systolic blood pressure < 90 mmHg, diastolic blood pressure < 60 mmHg, or heart rate >100 bpm) at screening
- Have clinically significant elevation of liver enzymes (> 3 times the upper limit of normal) or clinically relevant renal disease, (creatinine >1.5) or any other clinically significant abnormal laboratory test results found during medical screening as determined by the Principal Investigator
- Have a history of major mental illness that in the opinion of the Investigator may affect the ability of the subject to safely participate and reliably complete the study
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 |
|---|---|---|
| Opium Tincture USP DeodorizedTreatment A - 0.6 mL, MP-101 (10 mg/mL, Opium Tincture (OT)), USP (Deodorized) QID oral drops, followed by Treatment B - 0.6 mL, MP-101, 1/12 dilution, (0.833 mg/mL, OT), USP (Deodorized) QID oral drops, followed by Treatment C - 0.6 mL, MP-101 (10 mg/mL Opium Tincture with reduced uncharacterized material) QID oral drops | Drug | Group 1 |
| Opium Tincture USP DeodorizedTreatment B - 0.6 mL, MP-101, 1/12 dilution, (0.833 mg/mL, OT), USP (Deodorized) QID oral drops, followed by Treatment A - 0.6 mL, MP-101 (10 mg/mL, Opium Tincture (OT)), USP (Deodorized) QID oral drops, followed by Treatment C - 0.6 mL, MP-101 (10 mg/mL Opium Tincture with reduced uncharacterized material) QID oral drops | Drug | Group 2 |
Primary outcome
Percentage of responders between Treatments A and B for the Intent-To-Treat (ITT) population
Time frame: Last day of Periods 1 (Days 16-17) and 2 (Days 25-26)
30% or greater reduction in 24-hour stool volume from baseline
8 secondary outcomes
Percentage of responders between Treatments A and B for the Modified-Intent-To-Treat (mITT) population
Time frame: Last day of Periods 1 (Days 16-17) and 2 (Days 25-26)
Establish non-inferiority of the percentage of responders between Treatment A and Treatment C
Time frame: Last day of Period 3 (Day 34-35)
24-hour stool volume comparison between Treatment A and B for ITT and mITT
Time frame: Last day of Periods 1 (Day 16-17) and 2 (Day 25-26)
24-hour fecal events between Treatment A and B for ITT and mITT
Time frame: Period 1 (Day 10-16) and 2 (Day 19-25)
24-hour nocturnal fecal events between Treatment A and B for ITT and mITT
Time frame: Period 1 (Day 10-16) and 2 (Day 19-25)
Single and multiple dose Pharmacokinetic (PK) parameters
Time frame: Day-1, Day 1, Day 7, Day 57-59
Number of Participants with Adverse Events
Time frame: Up to 60 Days
Number of Participants Up-Titrated
Time frame: Days 35-60
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2015.
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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.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Opium Tincture USP Deodorized in people with Short Bowel Syndrome and Short Gut Syndrome, run by Marathon Pharmaceuticals, LLC at 5 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 19 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: withdrawn, as of the sponsor's last update on 18 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Percentage of responders between Treatments A and B for the Intent-To-Treat (ITT) population (time frame: Last day of Periods 1 (Days 16-17) and 2 (Days 25-26)). 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 18 Feb 2015.