ive GATTEX 0.05 mg/kg/day. Ninety-seven percent (76/78) of subjects from Study 1 elected to enroll in Study 2. An additional 12 subjects entered Study 2, who had been optimized and stabilized but not randomized in Study 1 because of closed enrollment. Of responders in Study 1 who entered Study 2, 100% (25/25) sustained their response to GATTEX after one year of continuous treatment. A 20% or greater reduction of parenteral support was achieved in 72% (31/43) of subjects after an additional 28 weeks of continuous GATTEX treatment. The mean reduction of weekly PN/I.V. volume was 5.2 L/week after one year of continuous GATTEX treatment. Six subjects in Study 2 were weaned off their PN/I.V. support while on GATTEX. Subjects were maintained on GATTEX even if no longer requiring PN/I.V. support. These 6 subjects had required PN/I.V. support for 3 to 18 years, and prior to GATTEX had required between 4 L/week and 13 L/week of PN/I.V. support.
Study 3 (Placebo-controlled) and Study 4 (Blinded uncontrolled extension of Study 3)
Study 3. Study 3 was a randomized, double-blind, placebo-controlled, three parallel-group, multinational study in adults with Short Bowel Syndrome who were dependent on parenteral nutrition/intravenous (PN/I.V.) support for at least 12 months and required PN at least 3 times per week. After a period of optimization and stabilization similar to Study 1, subjects were randomized to receive 24 weeks of one of the following treatment regimens: GATTEX 0.05 mg/kg/day (n=35), GATTEX 0.10 mg/kg/day dose (n=33), or placebo (n=16). The treatment groups were compared using the intent-to-treat population of this study which was defined as all randomized patients who were administered at least one dose of study drug. This population contained one less patient in the 0.10 mg/kg/day dose group hence n=32 in this group for all analyses. The primary efficacy endpoint was a graded categorical score that did not achieve statistical significance for the high dose. Further eva luation of PN/I.V. volume reduction using the endpoint of response (defined as at least 20% reduction in PN/I.V. fluid from Baseline to Weeks 20 and 24) showed that 46% of subjects on GATTEX 0.05 mg/kg/day responded versus 6% on placebo. Subjects on GATTEX at both dose levels experienced a 2.5 L/week reduction in parenteral support requirements versus 0.9 L/week for placebo at 24 weeks. Two subjects in the GATTEX 0.05 mg/kg/day dose group were weaned off parenteral support by Week 24.
Study 4. Study 4 was a blinded, uncontrolled extension of Study 3, in which 65 subjects from Study 3 received GATTEX for up to an additional 28 weeks of treatment. Of responders in Study 3 who entered Study 4, 75% sustained response on GATTEX after one year of treatment. In the GATTEX 0.05 mg/kg/day dose group, a 20% or greater reduction of parenteral support was achieved in 68% (17/25) of subjects. The mean reduction of weekly PN/I.V. volume was 4.9 L/week (52% reduction from baseline) after one year of continuous GATTEX treatment. The subjects who had been completely weaned off PN/I.V. support in Study 3 remained off parenteral support through Study 4. During Study 4, an additional subject from Study 3 was weaned off parenteral support.
How Supplied/Storage and Handling
How Supplied
GATTEX® (teduglutide [rDNA origin]) for injection is supplied in a sterile, single-use glass vial containing 5 mg of teduglutide as a white, lyophilized powder to be reconstituted with 0.5 mL Sterile Water for Injection. The product to be dispensed is eith |
