Adult Clinical Study Data

STEPS & STEPS-2 Trial

GATTEX Was Studied in Adult Patients With SBS-IF1

GATTEX was evaluated in STEPS, a phase 3, randomized, double-blind,
placebo-controlled trial, and STEPS-2, an open-label extension study1

Primary endpoint:
Defined as a ≥20% reduction in weekly PS volume from baseline (ie, immediately before randomization to Weeks 20 and 24 in STEPS, and start of GATTEX treatment to the last visit in STEPS-2).1,2

6 Months

STEPS Trial1*

GATTEX (n=43)

Placebo (n=43)

24 Months

STEPS-2 Trial1

GATTEX/GATTEX enrolled (n=37) completed (n=30)

Placebo/GATTEX enrolled (n=39) completed (n=29)
30 patients completed 30 months of treatment on GATTEX

Not treated/GATTEX enrolled (n=12) completed (n=6)

30 Months in Total

  • Efficacy analysis was performed by comparing the intent-to-treat population in the treatment group and placebo group, respectively1
    • PS volume adjustments (up to a 30% decrease) and clinical assessments were made at Weeks 2, 4, 8, 12, 20, and 24

*After randomization of the intent-to-treat population (N=86), 3 patients in the GATTEX arm and 4 in the placebo arm discontinued treatment. Additionally, 1 patient in the GATTEX arm was not treated due to a randomization error. This resulted in a total of 78 evaluable patients.3

STEPS-2 was a 24-month, open-label extension study measuring long-term response (n=37, GATTEX/GATTEX; n=39, placebo/GATTEX; n=12, not treated/GATTEX)2†

24-Month Extension Study

Enrolled in STEPS-2

GATTEX n=37
(0.05 mg/kg/day)

Placebo/GATTEX n=39
(0.05 mg/kg/day)

Not treated in STEPS/GATTEX n=12
(0.05 mg/kg/day)

Completed STEPS-2†

GATTEX n=30
(0.05 mg/kg/day) GATTEX=30 months

Placebo/GATTEX n=29
(0.05 mg/kg/day) GATTEX=24 months

Not treated in STEPS/GATTEX n=6
(0.05 mg/kg/day) GATTEX=24 months

  • Of the 78 patients who completed STEPS, 97% (76/78) elected to enroll in STEPS-22

  • All patients received GATTEX in STEPS-22

  • Efficacy analysis was calculated in patients who completed 24 months of treatment with GATTEX in the placebo/GATTEX subgroup, and 30 months of treatment with GATTEX in the GATTEX/GATTEX subgroup2

  • Of the 88 patients enrolled in STEPS-22:

    • 65 (74%) completed the study (n=30/37, GATTEX/GATTEX; n=29/39, placebo/GATTEX; n=6/12, not treated/GATTEX)2

*After randomization of the intent-to-treat population (N=86), 3 patients in the GATTEX arm and 4 patients in the placebo arm discontinued treatment. Additionally, 1 patient in the GATTEX arm was not treated due to a randomization error. This resulted in a total of 78 evaluable patients.3

†Patients who completed fluid optimization and stabilization but were not randomized in the initial 6-month, placebo-controlled study because of full study enrollment were eligible for direct enrollment into STEPS-2.2

GATTEX Was Studied in a Range of Adult Patients1,3

Patient baseline characteristics in STEPS Average Range
Age 50 years 18-82 years
Estimated small bowel length 77.3 cm 5-343 cm
Length of time on PS 6 years 1-26 years
Prescribed days per week on PS 5.73 days 3-7 days
Infusion volume 13 L/week 0.9-35 L/week

Intestinal Resection Baseline Characteristics

Patients had varying types of intestinal resection

%

(37/85)

of patients did not have colon-in-continuity.1 An average of 37% of the colons in these patients had been removed.4

%

(13/24)

of patients with an intact distal/terminal ileum had an ileocecal valve4

%

(38/85)

of patients had a stoma (most commonly jejunostomy and ileostomy)1

The most common reasons for intestinal resection included1:

  • Vascular disease: 34% (29/85)
  • Crohn’s disease: 21% (18/85)
  • Other: 21% (18/85)
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