Clinical Trial Data
A study was conducted to evaluate the efficacy, safety, and tolerability of GATTEX® (0.05 mg/kg per day vs. placebo) in PN-dependent adult patients with SBS.
Study of Teduglutide in PN-dependent Short Bowel Syndrome (STEPS)1
STEPS was a randomized, double-blind, placebo-controlled, parallel-group, multinational, multicenter clinical trial (27 sites) in adults with SBS who were dependent on parenteral nutrition/intravenous (PN/IV) support for at least 12 months and required parenteral support at least 3 times per week.**
PN/I.V. volume adjustments (up to 30% decrease) and clinical assessments were made at 2, 4, 8, 12, 20, and 24 weeks.
76 out of 78 patients who completed STEPS elected to continue in the open-label, 2-year extension study STEPS2.
- Additionally, 12 patients participated in STEPS2 who had been optimized and stabilized but not randomized in STEPS because of closed enrollment (n=88)
*STEPS is known as study 1 in the U.S. Prescribing Information.
**STEPS2 is known as study 2 in the U.S. Prescribing Information.
Characteristics associated with the population on GATTEX® in STEPS included adults:
• With or without colon in continuity
• With or without a stoma
• With dependency on parenteral support* ranging from 1 to 25 years
• With a mean range of remaining small intestine from 5 to 343 cm
* Parenteral support refers to parenteral nutrition and/or essential fluids.
1. GATTEX (teduglutide [rDNA origin]) [package insert]. Bedminster, NJ: NPS Pharmaceuticals, Inc. 2. Jeppesen PB, Pertkiewicz M, Messing B, et al. Teduglutide reduces need for parenteral support among patients with short bowel syndrome with intestinal failure. Gastroenterology. 2012;143(6):1473-1481.
Important Safety Information
Warnings and Precautions
Colorectal polyps were identified during clinical trials. There is a risk for acceleration of neoplastic growth. Colonoscopy of the entire colon with removal of polyps should be done within 6 months prior to starting treatment with GATTEX and is recommended after 1 year. Subsequent colonoscopies should be done as needed, but no less frequently than every 5 years. In case of intestinal malignancy (GI tract, hepatobiliary, pancreatic), discontinue GATTEX. The clinical decision to continue GATTEX in patients with non‑gastrointestinal malignancy should be made based on risk and benefit considerations.
Intestinal obstruction has been reported in clinical trials. In patients who develop obstruction, GATTEX should be temporarily discontinued pending further clinical evaluation and management.
Biliary and pancreatic disease
Cholecystitis, cholangitis, cholelithiasis, and pancreatitis have been reported in clinical trials. Patients should undergo laboratory assessment (bilirubin, alkaline phosphatase, lipase, amylase) before starting GATTEX. Subsequent laboratory tests should be done every 6 months. If clinically meaningful changes are seen, further evaluation is recommended including imaging, and continued treatment with GATTEX should be reassessed.
Fluid overload and congestive heart failure have been observed in clinical trials. There is potential for fluid overload while on GATTEX. If fluid overload occurs, especially in patients with underlying cardiovascular disease, parenteral support should be appropriately adjusted and GATTEX treatment reassessed.
Increased absorption of concomitant oral medication
Altered mental status in association with GATTEX has been observed in patients on benzodiazepines in clinical trials. Patients on concomitant oral drugs (e.g. benzodiazepines, phenothiazines) requiring titration or with a narrow therapeutic index may require dose adjustment while on GATTEX.
The most common adverse reactions (≥10%) across all studies with GATTEX are abdominal pain, injection site reactions, nausea, headaches, abdominal distension, upper respiratory tract infection. In addition, vomiting and fluid overload were reported in the SBS studies (1 and 3) at rates ≥10%.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You can also call Shire at 1‑855‑5GATTEX (1-855-542-8839).
GATTEX (teduglutide [rDNA origin]) for injection is indicated for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support.
For additional safety information, please click here for Prescribing Information