Safety, Efficacy and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome
Condition: Short Bowel Syndrome
MeSH terms: Short Bowel Syndrome; Syndrome
Intervention: Teduglutide; Standard Medical Therapy; Syringe; Needle
Intervention description: SC injection of 0.05 mg/kg teduglutide will be administered QD into abdomen or into either the thigh or arm for 24 weeks.; Standard medical therapy will be administered for 24 weeks.; Teduglutide will be administered using syringe (510k number: K980987).; Teduglutide will be administered using needle (510k number: K021475).
Intervention MeSH terms: Teduglutide
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 10.0
Sex: All
Minimum age: 4 Months
Maximum age: 12 Months
Healthy volunteers: No
Keywords: Short bowel syndrome; Teduglutide

Summary:

      The purpose of the study is to evaluate the safety, efficacy/pharmacodynamics (PD) and
      pharmacokinetics (PK) of teduglutide treatment in infants with short bowel syndrome (SBS)
      dependent on parenteral (PN) support.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Informed consent by the parent or legal guardian.

          -  Male or female infant 4 to 12 months corrected gestational age at screening.

          -  Weight at least 5 kilogram (kg) and weight-for-length Z-score greater than -2 at
             screening and baseline.

          -  Short bowel syndrome with dependence on parenteral support to provide at least 50% of
             fluid or caloric needs.

          -  Stable PN requirements for at least 1 month prior to screening, defined as a less than
             or equal to (<=) 10% change in the weight-normalized PN total fluid and caloric
             intake, despite attempts to wean PN, not withstanding transient instability for events
             such as sepsis or interruption of central venous access.

          -  Parent or legal guardian understands and is willing and able to fully adhere to study
             requirements as defined in this protocol.

        Exclusion Criteria:

          -  Previous treatment with teduglutide.

          -  Intestinal malabsorption due to a genetic condition, such as cystic fibrosis,
             microvillus inclusion disease, etc.

          -  Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe,
             active gastroschisis-related dysmotility, that is the primary contributing factor to
             feeding intolerance and inability to reduce PN support, prior to screening.
             Dysmotility is defined as severe if it is expected to limit the advancement of enteral
             feeding.

          -  Inability to advance oral or enteral feeding due to lack of access to the gut, such as
             oral aversion in the absence of a feeding tube.

          -  Intestinal obstruction or clinically significant intestinal stenosis.

          -  Major gastrointestinal surgical intervention, such as serial transverse enteroplasty
             or major intestinal resection or anastomosis, within 3 months prior to screening or
             planned during the study period.

          -  Unstable cardiac disease.

          -  Renal dysfunction, defined as estimated glomerular filtration rate less than (<) 50
             milliliter per minute (mL/min) per 1.73 square meter (m^2).

          -  Biliary obstruction, stenosis, or malformation.

          -  Clinically significant pancreatic disease.

          -  Severe hepatic dysfunction or portal hypertension, defined by at least 2 of the
             following parameters:

               1. International normalized ratio (INR) greater than (>) 1.5 not corrected with PN
                  vitamin K

               2. Platelet count <100×10^3/ microliter (mcL) due to portal hypertension

               3. Presence of clinically significant gastric or esophageal varices

               4. Documented cirrhosis

          -  Persistent cholestasis defined as conjugated bilirubin >4 milligram per deciliter
             (mg/dL) (>68 micromoles per liter [mcmol/L]) over a 2 week period.

          -  More than 3 serious complications of intestinal failure (example [e.g.],
             catheter-associated bloodstream infections, interruption of nutrition due to feeding
             intolerance, catheter-associated thrombosis, severe fluid or electrolyte disturbances)
             within 1 month prior to or during screening.

          -  A history of cancer or a known cancer predisposition syndrome, such as juvenile
             polyposis or Beckwith-Wiedemann syndrome, or first degree relative with early onset of
             gastrointestinal cancer (including hepatobiliary and pancreatic cancers).

          -  Concurrent treatment with glucagon-like peptide-1 (GLP-1); glucagon-like peptide-2
             (GLP-2); insulin-like growth factor-1 (IGF-1); growth hormone, somatostatin, or
             analogs of these hormones; or glutamine.

          -  Participation in a clinical study using an experimental drug within 3 months or 5.5
             half-lives of the experimental drug, whichever is longer.

          -  Known or suspected intolerance or hypersensitivity to the investigational product,
             closely-related compounds, or any of the stated ingredients.

          -  Any condition, disease, illness, or circumstance that, in the investigator's opinion,
             puts the participant at any undue risk, prevents completion of the study, or
             interferes with analysis of the study results.