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

NCT ID: NCT02242656

Last Updated: 2015-02-18

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE3
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-12-31

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Group 1

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

Group Type: EXPERIMENTAL

Opium Tincture USP Deodorized

Intervention Type: 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 2

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

Group Type: EXPERIMENTAL

Opium Tincture USP Deodorized

Intervention Type: DRUG

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

Interventions

Opium Tincture USP Deodorized

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

Intervention Type: DRUG

Opium Tincture USP Deodorized

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

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Be male or female adults, 18 years of age or older at the time of consent

2. 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

3. Have a history of persistent loose stools for more than 4 weeks

4. Be on a combination of Opium Tincture and an anti-diarrheal (loperamide or diphenoxylate) or an anti-diarrheal agent alone

5. 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

6. Be able to maintain their current diet for the duration of the study

7. Be on stable nutritional support (parenteral or oral)

8. 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)

9. Females of childbearing potential must agree to use 1 of the following acceptable birth control methods:

1. Surgically sterile (hysterectomy or bilateral oophorectomy)

2. Surgically sterile (bilateral tubal ligation with surgery at least 6 weeks prior to study initiation)

3. Intrauterine device (IUD) in place for at least 3 months

4. Abstinence (not having sexual intercourse)

5. Barrier method (condom or diaphragm) with spermicide for at least 14 days prior to screening and through study completion

6. Stable hormonal contraceptive for at least 3 months prior to study and through study completion

7. Vasectomized partner

10. Females of childbearing potential must have a negative serum human chorionic gonadotropin (hCG) pregnancy test at screening

11. Be able to understand and provide signed informed consent

Exclusion Criteria

1. Unable or unwilling to stop the use of Opium Tincture or any anti-diarrheal medication at the screening visit.

2. 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

3. 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

4. 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

5. 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

6. 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)

7. Have a known allergy or intolerance to Opium Tincture or any of the excipients in the formulation (alcohol, opium, or morphine)

8. 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

9. Are currently taking antibiotics for bacterial overgrowth

10. Have participated in another interventional clinical trial within 30 days prior to screening with the exception of observational cohort studies or non-interventional studies.

11. Have known or suspected pregnancy, planned pregnancy, or lactation

12. Have a planned surgery over the course of the study

13. 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Marathon Pharmaceuticals, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Northwestern University Feinberg School of Medicine

Chicago, Illinois, United States

Indiana University

Indianapolis, Indiana, United States

Cleveland Clinic

Cleveland, Ohio, United States

Regional Infectious Diseases Infusion Center

Lima, Ohio, United States

Vanderbilt Center for Human Nutrition

Nashville, Tennessee, United States

Countries

United States

Other Identifiers

MP-101-CL-001

Identifier Type: -

Identifier Source: org_study_id