MedPath

Efficacy and Safety Trial of Elobixibat in Patients With Chronic Idiopathic Constipation

Phase 3
Terminated
Conditions
Chronic Idiopathic Constipation
Interventions
Registration Number
NCT01833065
Lead Sponsor
Ferring Pharmaceuticals
Brief Summary

12 Week Efficacy and Safety Trial Followed by a 4 Week Withdrawal Period for Patients with Chronic Idiopathic Constipation.

Detailed Description

The present trial was designed to determine the efficacy and safety of elobixibat treatment (at both doses of 5 mg and 10 mg/day) compared to placebo treatment for 12-week Treatment Period followed by a 4-week Withdrawal Period in patients with chronic idiopathic constipation. During Withdrawal Period a sub-group of patients in elobixibat 5 mg and 10 mg treatment arms respectively received placebo treatment, while rest of the patients continued with 5 mg and 10 mg treatment in respective groups. In placebo groups, patients received elobixibat 10 mg treatment during Withdrawal Period. Patients were followed-up for 2 weeks after end of the Withdrawal Period.

The assessment of primary and key secondary end points was done for patients who completed the first 12 weeks of treatment period. Incidence of Adverse Events (AEs) were reported till 2 weeks after end of the Withdrawal Period.

The trial was early terminated due to a distribution issue with the trial medication.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
314
Inclusion Criteria
  • Body mass index (BMI) ≥18.5 but <35.0 kg/m^2

  • Male or female ≥18 years of age

  • Reports <3 spontaneous Bowel Movements (BM) per week and reports one or more of the following symptoms for the last 3 months with symptom onset at least 6 months before the Screening Visit or before starting chronic therapy with any laxative:

    1. Straining during at least 25% of defecations
    2. Lumpy or hard stools during at least 25% of defecations
    3. Sensation of incomplete evacuation during at least 25% of defecations
  • Is ambulatory and community dwelling

  • An initial colonoscopy is required if recommended by national guidelines

Exclusion Criteria
  • Reports loose (mushy) or watery stools in the absence of any laxative intake in the form of a tablet, a suppository or an enema, or prohibited medicine for >25% of BMs
  • The patient reports a BSFS of 6 or 7 during the Pretreatment Period
  • Has irritable bowel syndrome (IBS) with pain/discomfort as predominant symptoms
  • Has a structural abnormality of the Gastrointestinal (GI) tract or a disease or condition that can affect GI motility
  • Has a history of diverticulitis, chronic pancreatitis, active peptic ulcer disease (PUD) not adequately treated, ischaemic colitis, inflammatory bowel disease, laxative abuse, faecal impaction that required hospitalization or emergency treatment, pseudo-obstruction, megacolon, megarectum, bowel obstruction, descending perineum syndrome, ovarian cysts, endometriosis, solitary rectal ulcer syndrome, systemic sclerosis, pre-malignant colonic disease (e.g., familial adenomatous polyposis or hereditary non-polyposis colorectal cancer) or other forms of familial colorectal cancer
  • Has unexplained and clinically significant GI alarm signals (e.g., lower GI bleeding or heme-positive stool in the absence of known internal or external haemorrhoids, iron-deficiency anaemia, unexplained weight loss) or systemic signs of infection or colitis
  • Has a potential central nervous system (CNS) cause of constipation (e.g., Parkinson's disease, spinal cord injury, multiple sclerosis)
  • Has intestinal/rectal prolapse or other known pelvic floor dysfunction
  • Commonly uses digital maneuvers (perianal pressure or digital disimpaction) or vaginal splinting to facilitate the passage of a bowel movement
  • Has a history of diabetic neuropathy
  • Has a history of bariatric surgery for treatment of obesity; surgery to remove a segment of the GI tract; or surgery of the abdomen, pelvic or retroperitoneal area during the 6 months prior to Screening; or appendectomy or cholecystectomy 3 months prior to screening; or other major surgery 1 month prior to Screening
  • Has a history of cancer with last date of proven disease activity/presence of malignancy within 5 years, except for adequately treated basal cell carcinoma of the skin, cervical dysplasia, or carcinoma in situ of the skin or the cervix
  • Known human immunodeficiency virus (HIV) or Hepatitis B/C (HBV/HCV) infection
  • Has a history of hospitalization for any psychiatric disorder, or any suicide attempt in the 2 years prior to Screening
  • Is actively abusing alcohol or drugs or has a history of alcohol or drug abuse during the 6 months prior to Screening
  • Is being treated for hypothyroidism, but the dose of medication has not been stable for at least 3 months at the time of Screening
  • Is a pregnant, breast-feeding, or lactating woman

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
EBX 5Elobixibat 5 mg/dayElobixibat 5 mg/day
EBX 10Elobixibat 10 mg/dayElobixibat 10 mg/day
PLCBOPlaceboPlacebo
Primary Outcome Measures
NameTimeMethod
Overall Complete Spontaneous Bowel Movement (CSBM) ResponseDuring the first 12 weeks

This outcome measured the percentage of patients who were CSBM responders. A CSBM responder was defined as a patient with ≥3 CSBMs per week and an increase of ≥1 CSBM per week from Baseline, for at least 9 of the 12 weeks in the 12-week Treatment Period, including at least 3 weeks during Weeks 9-12.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in Weekly Frequency of Spontaneous Bowel Movement (SBMs)From Baseline (2-week Pretreatment Period) to overall first 12-weeks of Treatment Period

The change from Baseline for the continuous variable was estimated using a repeated measures analysis of covariance (ANCOVA) model.

Change From Baseline in Weekly Stool Consistency of SBMsFrom Baseline (2-week Pretreatment Period) to overall first 12-weeks of Treatment Period

The stool consistency is measured using the seven-point ordinal Bristol Stool Form Scale (BSFS) score. The BSFS classifies human stool into seven types and points them accordingly.

Type 1: Separate hard lumps, like nuts (hard to pass) Type 2: Sausage-shaped, but lumpy Type 3: Like a sausage but with cracks on its surface Type 4: Like a sausage or snake, smooth and soft Type 5: Soft blobs with clear cut edges (passed easily) Type 6: Fluffy pieces with ragged edges, a mushy stool Type 7: Watery, no solid pieces, entirely liquid Types 1 and 2 indicate constipation, with 3 and 4 represents the ideal stool form (especially the latter), and 5, 6 and 7 tends towards diarrhoea .

For a given assessment week, the weekly stool consistency was defined as the sum of non-missing stool consistency score for SBMs during that week divided by the number of non-missing stool consistency score for SBMs during that week. The parameter was analysed using repeated measures ANCOVA model.

Occurrence of CSBM ResponseWithin first 24 hours of treatment initiation

This outcome measured the percentage of patients who had a CSBM within 24 hours after the first dose of treatment. A CSBM was defined as a spontaneous (occurring without laxative within the preceding 24 hours, including no rescue medication within the preceding 24 hours) bowel movement (as interpreted by the patient, with a beginning and an end, including single or multiple stools), accompanied by a patient reported sense of complete evacuation ('complete').

Change From Baseline in Weekly Abdominal Discomfort ScoreFrom Baseline (2-week Pretreatment Period) to overall first 12-weeks of Treatment Period

The abdominal discomfort score was measured using the five-point ordinal scale (1=None, 2=Mild, 3=Moderate, 4=Severe, and 5=Very severe).

For a given assessment week, the weekly abdominal discomfort score was defined as the sum of non-missing abdominal discomfort score for SBMs during that week divided by the number of non-missing abdominal discomfort score for SBMs during that week. The parameter was analysed using repeated measures ANCOVA model.

Change From Baseline in Weekly Degree of Straining of SBMsFrom Baseline (2-week Pretreatment Period) to overall first 12-weeks of Treatment Period

The degree of straining was measured using the five-point ordinal scale (1=Not at all, 2=A little bit, 3=A moderate amount, 4=A great deal, and 5=An extreme amount).

For a given assessment week, the weekly degree of straining was defined as the sum of non-missing straining score for SBMs during that week divided by the number of non-missing straining score for SBMs during that week. The parameter was analysed using repeated measures ANCOVA model.

Change From Baseline in Weekly Abdominal Bloating ScoreFrom Baseline (2-week Pretreatment Period) to overall first 12-weeks of Treatment Period

The abdominal pain score was measured using the five-point ordinal scale (1=None, 2=Mild, 3=Moderate, 4=Severe, and 5=Very severe).

For a given assessment week, the weekly abdominal bloating score was defined as the sum of non-missing abdominal bloating score for SBMs during that week divided by the number of non-missing abdominal bloating score for SBMs during that week. The parameter was analysed using repeated measures ANCOVA model.

Total Patient Assessment of Constipation - Quality of Life (PAC-QOL) Score ResponderAt Week 12

This outcome measured the percentage of patients who were PAC-QOL score responder at 12-week Treatment Period. A PAC-QOL score responder was defined as a patient with ≥50% reduction in total PAC-QOL score from Baseline at Week 12.

PAC-QOL is a 28-item questionnaire for psychometric assessment of disease-specific quality of life. The questionnaire is based on 5-point Likert scale; ranging from 0 \[none of the time or not at all\] to 4 \[all of the time or extremely\]). A lower score indicates a better Quality of Life. The PAC-QOL questionnaire is developed specifically for patients with constipation.

Total PAC-QOL score was averaged from the individual item score.

Trial Locations

Locations (92)

David Geffen School of Medicine at University of California, Los Angeles

🇺🇸

Los Angeles, California, United States

Zasa Clinical Research

🇺🇸

Boynton Beach, Florida, United States

Meridien Research

🇺🇸

Bradenton, Florida, United States

Health Awareness, Inc.

🇺🇸

Jupiter, Florida, United States

Gastrocentro Carioca Centro Gast e Endosc Dig, Ltda

🇧🇷

Rio de Janeiro, Brazil

Hospital Israelita Albert Einstein

🇧🇷

São Paulo, Brazil

Dr Anil K Gupta Medicine Professional Corp.

🇨🇦

Toronto, Ontario, Canada

Toronto Digestive Diseases Associates, Inc.

🇨🇦

Toronto, Ontario, Canada

Karolinska University Hospital Huddinge

🇸🇪

Stockholm, Sweden

Clinfan Szolgáltató Kft

🇭🇺

Szekszárd, Hungary

Fundacao IMEPEM - Universidade Federal de Juiz de Fora

🇧🇷

Juiz de Fora, Minas Gerais, Brazil

Dr. Zubar Fazel Vawda

🇿🇦

Durban, KwaZulu-Natal, South Africa

Heartland Research Associates, LLC

🇺🇸

Wichita, Kansas, United States

Professional Research Network of Kansas, LLC

🇺🇸

Witchita, Kansas, United States

Louisiana Research Center, LLC

🇺🇸

Shreveport, Louisiana, United States

Fakultní Nemocnice Ostrava

🇨🇿

Ostrava, Severomoravsky Kraj, Czech Republic

New Phase Research and Development, LLC

🇺🇸

Knoxville, Tennessee, United States

Preferred Research Partners

🇺🇸

Little Rock, Arkansas, United States

Palmetto Clinical Research

🇺🇸

Summerville, South Carolina, United States

Genova Clinical Research, Inc.

🇺🇸

Tucson, Arizona, United States

Arkansas Gastroenterology

🇺🇸

North Little Rock, Arkansas, United States

Synexus Merseyside Clinical Research Centre

🇬🇧

Liverpool, England, United Kingdom

Anderson Gastroenterology Associates

🇺🇸

Anderson, South Carolina, United States

Gastroenterologicka a interni ambulance

🇨🇿

Ceské Budejovice, Czech Republic

SPZOZ Szpital Kliniczny nr 1 im. Norberta Barlickiego Uniwersytetu Medycznego w Lodzi

🇵🇱

Lódz, Lodzkie, Poland

Medica Pro Familia Sp. z o.o. S.K.A.

🇵🇱

Warszawa, Mazowieckie, Poland

Semmelweis Egyetem

🇭🇺

Budapest, Hungary

Gastroenterology Associates of Fairfield County

🇺🇸

Bridgeport, Connecticut, United States

Krakowskie Centrum Medyczne Sp. z o.o.

🇵🇱

Kraków, Malopolskie, Poland

Lama Medical Care s.r.o., Gastroentero-hepatologicke centrum Thalion

🇸🇰

Bratislava, Slovakia

Gastro I.s.r.o.

🇸🇰

Prešov, Slovakia

GEA s.r.o Gastroenterologicka ambulancia

🇸🇰

Trnava, Slovakia

JOSHA Research

🇿🇦

Bloemfontein, Free State, South Africa

Pannónia Magánorvosi Centrum Kft.

🇭🇺

Budapest, Hungary

Synexus Lancashire Clinical Research Centre

🇬🇧

Chorley, England, United Kingdom

HCCA Clinical Research Solutions

🇺🇸

Jackson, Tennessee, United States

Associates in Gastroenterology, LLC

🇺🇸

Hermitage, Tennessee, United States

Miskolci Semmelweis Kórház és Egyetemi Oktatókórház

🇭🇺

Miskolc, Hungary

Medicor Centrum Medyczne Tadeusz Mazurek

🇵🇱

Rzeszów, Podkarpackie, Poland

Be Part Yoluntu Centre

🇿🇦

Paarl, Western Cape, South Africa

Hospital Centro Internacional de Medicina Chihuahua

🇲🇽

Chihuahua, Mexico

Louis Leipoldt Medi-Clinic Medical Centre

🇿🇦

Bellville, Western Cape, South Africa

Jahn Ferenc Dél-Pesti Kórház és Rendelointézet

🇭🇺

Budapest, Hungary

Synexus Thames Valley Clinical Research Centre

🇬🇧

Reading, England, United Kingdom

West Gastroenterology Associates

🇺🇸

Los Angeles, California, United States

Sanitas Research

🇺🇸

Coral Gables, Florida, United States

Southeast Clinical Research, LLC

🇺🇸

Jacksonville, Florida, United States

Lake Internal Medicine Associates

🇺🇸

Eustis, Florida, United States

Health Care Family Rehab Corp.

🇺🇸

Hialeah, Florida, United States

Southeast Regional Research Group

🇺🇸

Columbus, Georgia, United States

Mount Vernon Clinical Research

🇺🇸

Atlanta, Georgia, United States

Premier Healthcare Research, LLC

🇺🇸

Evanston, Illinois, United States

Beacon Clinical Research, LLC

🇺🇸

Brockton, Massachusetts, United States

Rockford Gastroenterology Associates, Ltd.

🇺🇸

Rockford, Illinois, United States

Peters Medical Research, LLC

🇺🇸

High Point, North Carolina, United States

Long Island Gastrointestinal Research Group

🇺🇸

Great Neck, New York, United States

PharmQuest, LLC

🇺🇸

Greensboro, North Carolina, United States

Carolina Digestive Health Associates, PA

🇺🇸

Concord, North Carolina, United States

Hometown Urgent Care and Occupational Health

🇺🇸

Dayton, Ohio, United States

Family Practice Center of Wadsworth

🇺🇸

Wadsworth, Ohio, United States

Sunstone Medical Research, LLC

🇺🇸

Medford, Oregon, United States

Family Medical Associates

🇺🇸

Levittown, Pennsylvania, United States

Research Across America

🇺🇸

Katy, Texas, United States

Gastroenterology Associates of Tidewater

🇺🇸

Chesapeake, Virginia, United States

Medical Arts Health Research Group

🇨🇦

Penticton, British Columbia, Canada

London Road Diagnostic Clinic and Medical Centre

🇨🇦

Sarnia, Ontario, Canada

Pro-Recherche Polyclinique des Ponts

🇨🇦

Saint Romuald, Quebec, Canada

Synexus Clinical Research GmbH

🇩🇪

Görlitz, Sachsen, Germany

CRU Hungary Kft.

🇭🇺

Szikszó, Hungary

Pándy Kálmán Megyei Kórház

🇭🇺

Gyula, Bekes, Hungary

Jávorszky Ödön Kórház

🇭🇺

Vác, Hungary

Vasútegészségügyi Nonprofit Kiemelten Közhasznú Kft

🇭🇺

Debrecen, Hungary

Borsod Abaúj Zemplén Megyei Kórház és Egyetemi Oktató Kórház

🇭🇺

Miskolc, Hungary

Accelerium Clinical Research

🇲🇽

Monterrey, Nuevo Leon, Mexico

Centro de Investigación Médico Biológica y Terapia Avanzada SC

🇲🇽

Guadalajara, Jalisco, Mexico

Medical Care and Research

🇲🇽

Mérida, Yucatan, Mexico

Przychodnia Polskiej Fundacji Gastroenterologii Filia Nr 1 NZOZ

🇵🇱

Warszawa, Mazowieckie, Poland

Szpital Uniwersytecki nr 2 im. dr. Jana Biziela w Bydgoszczy

🇵🇱

Bydgoszcz, Kujawsko-pomorskie, Poland

Indywidualna Specjalistyczna Praktyka Lekarska Adam Kopon

🇵🇱

Torun, Kujawsko-pomorskie, Poland

KM Management sro

🇸🇰

Nitra, Slovakia

PIGEAS s.r.o.

🇸🇰

Martin, Slovakia

Synexus Clinical Research SA

🇿🇦

Pretoria, Gauteng, South Africa

Mzansi Ethical Research Centre

🇿🇦

Middelburg, Mpumalanga, South Africa

Sahlgrenska University Hospital

🇸🇪

Gothenburg, Sweden

Uppsala Akademiska Sjukhus

🇸🇪

Uppsala, Sweden

Synexus Scotland Clinical Research Centre

🇬🇧

Glasgow, Scotland, United Kingdom

Wake Research Associates, LLC

🇺🇸

Raleigh, North Carolina, United States

Birmingham Gastroenterology Associates, PC

🇺🇸

Birmingham, Alabama, United States

Sacramento Research Medical Group

🇺🇸

Sacramento, California, United States

Precision Research Institute, LLC

🇺🇸

San Diego, California, United States

Quality Clinical Research, Inc.

🇺🇸

Omaha, Nebraska, United States

Clinical Research Associates, LLC

🇺🇸

Oklahoma City, Oklahoma, United States

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