NPS initiates US Gattex trial in SBS

NPS Pharmaceuticals, a US specialty drug company focused on developing therapeutics for rare gastrointestinal and endocrine disorders, has begun patient enrollment in a Phase III registrational study of Gattex (teduglutide) in short-bowl syndrome patients.

The international, double-blind, placebo-controlled safety and efficacy study known as STEPS will enroll approximately 86 parenteral nutrition-dependent SBS patients. The trial includes an initial three- to eight-week PN optimization and stabilization period, after which patients will be randomized 1:1 to compare daily subcutaneous dosing of 0.05mg/kg of Gattex to placebo over a 24-week treatment period. The primary efficacy endpoint is the percentage of patients who achieve a 20% or greater reduction in weekly PN volume at week 20 and maintain that response at week 24, when compared to baseline. The study's secondary objectives will evaluate efficacy variables based on reductions in PN volume or the direct effects of improved intestinal absorption of fluid. These variables include: duration of response; the proportion of patients with a 20% or greater reduction or a two liter or greater reduction from baseline in weekly PN at week 20 and maintained through week 24; the number of patients who discontinue PN, including the time of discontinuation; and the absolute and percentage change in PN.

"We are advancing STEPS with the support of our partner Nycomed and expect to complete patient enrollment in approximately 12 to 15 months," said NPS chief executive Francois Nader.