Study of Teduglutide Effectiveness in Parenteral Nutrition (PN)-Dependent Short Bowel Syndrome (SBS) Subjects

Study Overview
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Brief Summary
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Teduglutide is an investigative medicine being evaluated as a possible treatment for people with parenteral nutrition (PN) dependent Short Bowel Syndrome (SBS). Teduglutide is similar to a protein the body makes. When people have SBS, their bodies do not make enough of the protein and they have trouble getting nutrients and fluids from the food they eat and drink. This study was designed to provide evidence of efficacy, safety, and tolerability of teduglutide 0.05 mg/kg daily in SBS subjects.

Official Title
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A 24-Week Study of the Efficacy and Safety of Teduglutide in Subjects With Parenteral Nutrition-Dependent Short Bowel Syndrome

Conditions
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Short Bowel Syndrome

Intervention / Treatment
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* Drug: teduglutide
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed and dated informed consent prior to any study-related procedures are performed Men and women 18 years of age or older at the time of informed consent signing Intestinal failure resulting in Short Bowel Syndrome At least 12 months of continuous PN dependency 12 weeks of clinical remission of Crohn's disease (CD) prior to dosing PN required at least 3 times weekly A stable PN volume for four weeks prior to dosing Exclusion Criteria: History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state Participation in clinical study within 30 days for drug or 90 days for antibody Use of native GLP-2 or human growth hormone (HGH) within 6 months of screening Use of iv glutamine within 30 days prior to screening Use of teduglutide CD patients who have been treated with biological therapy within 6 months of screening IBD patients who require chronic systemic immunosuppressant therapy More than 4 SBS- or PN-related hospitalizations within 12 months of screening Unplanned hospitalization within one month of screening Pregnant or lactating women Body weight > 88kg Body mass index (BMI) < 15 kg/m2 Severe hepatic impairment or disturbed renal function Female subjects who are not surgically sterile or postmenopausal or who are not using medically acceptable methods of birth control during and for 30 days after the treatment period Not capable of understanding or not willing to adhere to the study visit schedules and other protocol requirements Any condition or circumstance that is the investigator's opinion would put the subject at any undue risk, prevent completion of the study, or interfere with the analysis of the study results Significant active, uncontrolled, untreated systemic diseases

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Teduglutide<br>0.05 mg/kg/day sc dose of teduglutide | Drug: teduglutide<br>* 0.05 mg/kg/day sc injection for 24 weeks<br>* Other names: ALX-0600;|
| Placebo Comparator: Placebo<br>Matching subcutaneous dose of placebo to teduglutide | Drug: placebo<br>* Matching daily subcutaneous dose of placebo to teduglutide for 24 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Responder | Comparison of subjects treated with teduglutide to placebo who achieve a 20 to 100% reduction from baseline in weekly parenteral nutrition/intravenous fluid (PN/I.V.) volume at weeks 20 and 24. | Weeks 20 and 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Absolute Change in PN/I.V. Volume From Baseline to Last Time Point | Absolute change in the volume of PN/I.V. from baseline (Week 0) to the visit when the last data point was collected (week 4 through week 24, or earlier if the subject discontinued early). | Week 0 to last visit when data was collected. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SBS, short bowel syndrome, TPN, parenteral nutrition, PN