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

NCT02242656 | Withdrawn Phase 3

• 1 other identifier: MP-101-CL-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 5

Brief 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.

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; loperamide; diphenoxylate; ileostomies; tincture of opium; DTO; codeine; morphine; opiate

Outcome Measures

Primary Outcomes (1)

• 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

  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

  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

  Last day of Period 3 (Day 34-35)

• 24-hour stool volume comparison between Treatment A and B for ITT and mITT

  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

  Period 1 (Day 10-16) and 2 (Day 19-25)

• 24-hour nocturnal fecal events between Treatment A and B for ITT and mITT

  Period 1 (Day 10-16) and 2 (Day 19-25)

Study Arms (2)

Group 1

EXPERIMENTAL

Assigned to receive Treatment A (Period 1), Treatment B (Period 2), Treatment C (Period 3)

Drug: Opium Tincture USP Deodorized

Group 2

EXPERIMENTAL

Assigned to receive Treatment B (Period 1), Treatment A (Period 2), Treatment C (Period 3)

Drug: Opium Tincture USP Deodorized

Interventions

Opium Tincture USP Deodorized DRUG

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

Group 1

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• a. 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
• 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

You may not qualify if:

• 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)
• 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

Sponsors & Collaborators

• Marathon Pharmaceuticals, LLC: lead

Study Sites (5)

Northwestern University Feinberg School of Medicine

Chicago, Illinois, 60611, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Regional Infectious Diseases Infusion Center

Lima, Ohio, 45801, United States

Location

Vanderbilt Center for Human Nutrition

Nashville, Tennessee, 37212-1150, United States

Location

MeSH Terms

Conditions

Short Bowel Syndrome

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2014
• First Posted: September 17, 2014
• Study Start: December 1, 2014
• Primary Completion: December 1, 2017
• Last Updated: February 18, 2015

Record last verified: 2014-10

Locations

US (5)