The international, double-blind, placebo-controlled safety and efficacy study of Gattex is known as STEPS. STEPS will enroll approximately 86 parenteral nutrition (PN)-dependent short-bowl syndrome (SBS) patients in North America and Europe.
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. STEPS was designed with input from the FDA and NPS believes positive results will enable it to seek US marketing approval for Gattex for patients with PN-dependent SBS.
Francois Nader, president and CEO of NPS Pharmaceuticals, said: Launching STEPS is an important milestone for NPS, bringing us closer to our goal of introducing a new standard of care to reduce PN dependence for SBS patients. We are advancing Steps with the support of our partner Nycomed and expect to complete patient enrollment in approximately 12 to 15 months.