DRUG
Placebo
placebo injectable subcutaneously daily into thigh or abdomen
Status
Completed
Phase
Phase 3
Enrollment
84
Locations
32
Results
Posted
Publications
6
Study summary
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of teduglutide compared with placebo in subjects with parenteral nutrition (PN)-dependent short bowel syndrome (SBS).
Teduglutide is an analog of glucagon-like peptide 2 (GLP-2), a naturally occurring hormone that regulates the growth, proliferation, and maintenance of cells lining the gastrointestinal tract. Teduglutide has been shown in animal studies and previous human clinical trials to increase the size and number of these cells, thereby increasing the absorptive surface area of the intestines. The multicenter, double-blind, international Phase III trial will randomly assign approximately 80 patients to receive daily subcutaneous injections of 0.05 milligrams or 0.10 milligrams of teduglutide per kilogram of body weight, or a placebo. Dosing will continue for a period of six months. The primary endpoint in the study is a reduction in the use of intravenous feeding, which is often required to sustain life in patients with SBS.
Interventions
DRUG
placebo injectable subcutaneously daily into thigh or abdomen
DRUG
Teduglutide 0.05 mg/kg/d daily injectable subcutaneously into the thigh or abdomen
DRUG
Teduglutide 0.1 /g/kg/d daily injection subcutaneously into thigh or abdomen
Timeline
First posted
Apr 14, 2004
Study start
May 25, 2004
Primary completion
Jul 6, 2007
Study completion
Jul 6, 2007
Results posted
Jul 15, 2013
Registry updated
Jun 9, 2021
Outcomes
A Graded Response Score in Parenteral Nutrition (PN) Reduction
Time frame · 6 months
The intensity of the response relied on a reduction from Baseline in weekly parenteral nutrition (PN) volume (minimum reduction of 20% and a maximum of 100%). Duration of the response incorporated responses at Weeks(Wk) 16-20 and at Wk20-24. Zero (0 - lowest) assigned if \<20% reduction at Wk20-24 and reduction at Wk16-20 of \< 20%, 20-39%, or \>=40%. One (1) assigned if reduction of 20-39% at Wk20-24 but \< 20% at Wk16-20. Two(2) assigned if reductions of 40-99% at Wk20-24 AND \<20% at Wk16-20 OR 20-39% at Wk20-24 AND 20-39% at Wk16-20. Three (3) assigned if reductions of 100% at Wk20-24 AND \<20% at Wk16-20 OR 40-99% at Wk20-24 AND 20-39% at Wk16-20 OR 20-39% at Wk20-24 AND \>=40% at Wk16-20. Four (4) assigned if reductions of 100% at Wk20-24 AND 20-39% at Wk16-20 OR 40-99% at Wk20-24 AND \>=40% at Wk16-20.
Number of Subjects Achieving Binary Response at Week 20, Maintained at Week 24
Time frame · 6 months of treatment
An efficacy responder was defined as achieving at least a 20% reduction from Baseline to Week 20 and maintained at Week 24 in weekly actual PN infusion volume.
Eligibility
Inclusion Criteria: * Men and women, aged 18 years of age or older at the time of signing the informed consent form (ICF) * SBS as a result of major intestinal resection resulting in at least 12 months intravenous feeding * Body weight must be less than 90 kg * At baseline, subjects must require PN treatment to meet their caloric or electrolyte needs due to ongoing malabsorption at least 3 times weekly and to be on a stable PN regimen for 4 weeks before dosing * Body mass index (BMI) 18 to 27 kg/m2 * Adequate hepatic and renal function Exclusion Criteria: * History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state * History of alcohol or drug abuse (within previous year) * Participation in a clinical study within 30 days prior to signing the ICF, or concurrent participation in any clinical study * Clinically significant laboratory abnormalities at the time of randomization * Previous use of teduglutide (ALX-0600) * Prior use of native GLP-2 within 3 months of screening visit * Hospital admission within 1 month prior to screening visit * Pregnant or lactating women * Any condition or circumstance, which in the investigator's opinion would put the subject at any undue risk, prevent completion of the study, or interfere with analysis of the study results. * Presence of excluded disease: Radiation enteritis, Scleroderma, Celiac disease, Refractory/Tropical sprue, Pseudo-obstruction, Active inflammatory bowel disease (IBD), Pre-malignant/malignant change in colonoscopy biopsy or polypectomy, Surgery scheduled within the time frame of the study, Human immunodeficiency virus (HIV) positive test, Immunological disorders, Possible allergies to teduglutide or its constituents, Significant, active, uncontrolled, untreated systemic diseases
Study locations
Belgium · Canada · Denmark · France · Germany · Netherlands · Poland · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
Mayo Clinic Scottsdale
Scottsdale, Arizona, United States
Georgetown University
Washington D.C., District of Columbia, United States
Emory University Hospital
Atlanta, Georgia, United States
Northwestern Center for Clinical Research
Chicago, Illinois, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Albany Medical Center
Albany, New York, United States
Mount Sinai School of Medicine
New York, New York, United States
University of Rochester Medical Center
Rochester, New York, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, United States
University of Pennsylvania - Penn Nursing
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Rhode Island Hospital
Providence, Rhode Island, United States
Medical University of South Carolina
Charleston, South Carolina, United States
l'Hôpital Erasme
Brussels, Belgium
Royal Alexandra Hospital
Edmonton, Alberta, Canada
St. Paul's Hospital
Vancouver, British Columbia, Canada
St. Michael's Hospital
Toronto, Ontario, Canada
Toronto General Hospital
Toronto, Ontario, Canada
Rigshospitalet, University of Copenhagen
Copenhagen, Denmark
Hôpital Claude-Huriez
Lille, France
Hôpital de la Croix-Rousse
Lyon, France
Hôpital Edouard Herriot
Lyon, France
Hôpital Beaujon
Paris, France
Publications
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