Study of Teduglutide Effectiveness in Parenteral Nutrition (PN)-Dependent Short Bowel Syndrome (SBS) Subjects
Condition: Short Bowel Syndrome
MeSH terms: Short Bowel Syndrome; Syndrome
Intervention: teduglutide; placebo
Intervention description: 0.05 mg/kg/day sc injection for 24 weeks; Matching daily subcutaneous dose of placebo to teduglutide for 24 weeks
Intervention MeSH terms: Teduglutide
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 86.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: SBS; short bowel syndrome; TPN; parenteral nutrition; PN

Summary:

      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.
    

Eligibility criteria:

        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