Pediatric Clinical Study Data

Pivotal Study of GATTEX in Pediatric Patients1,2

GATTEX was evaluated in a 6-month, multicenter, phase 3 clinical study of pediatric patients aged 1 through 17 years who were dependent on PS (n=59)1,2

Primary endpoint: reduction in PS volume of ≥20% from baseline to the end of treatment2

  • Additional endpoints: reduction in PS volume from baseline and infusion hours/day, mean change in baseline PS infusion days/week at Week 24, and enteral autonomy2*

*Enteral autonomy: the ability to receive all nutrients, fluids, and calories through the GI tract without relying on PS.3

Study Design2

Screening
Period

Caregivers decided if patients received either:

GATTEX (n=50) or
standard of care (SOC) (n=9)

Randomized into
0.025 mg/kg/day (n=24)
and 0.05 mg/kg/day (n=26)

Enrolled and Completed
Study: 6 Months

GATTEX + standard of care (SOC)
0.025 mg/kg/day (n=24)

GATTEX + standard of care (SOC)
0.05 mg/kg/day (n=26)

Standard of care (SOC) only
n=9

Follow-Up Period:
4 Weeks

Standard of care (SOC)

PS requirements at baseline in the 6-month study1,3:

  • Stable PS support (defined as the inability to significantly reduce PS support [ie, 10% or less change in PS or advance in feeds]) for ≥3 months prior to and during screening as assessed by the investigator3
  • PS and specialized enteral nutrition were recorded in intake diaries by the child’s parent or guardian1,2
  • Patients had SBS resulting from intestinal resection and were dependent on PS that provided >30% of caloric and/or fluid/electrolyte needs3

GATTEX Was Studied in a Range of Pediatric Patients1,2

Patient baseline characteristics

Intent-to-treat population GATTEX 0.05 mg/kg/day
(n=26)
Demographics
Mean age, years (standard deviation [SD]) 6 (4)
1-11 years, n (%) 24 (92)
12-16 years, n (%) 2 (8)
17-<18 years, n (%) 0
Male, n (%) 19 (73)
Primary causes of SBS, n (%)
Gastroschisis 14 (54)
Midgut volvulus 6 (23)
Necrotizing enterocolitis 3 (12)
Intestinal atresia 1 (4)
Hirschsprung’s disease 1 (4)
Other 1 (4)
Baseline characteristics
Mean remaining small intestine length, cm (SD) 47 (28)
Stoma, n (%) Jejunostomy 5 (19) 4 (80)
Patients with remaining colon, n (%) Colon-in-continuity 25 (96) 22 (88)
Mean actual PS volume, mL/kg/day (SD) 60 (29)
Mean PS infusion time, days/week (SD) 7 (1)
Mean PS infusion time, hours/day (SD) 11 (3)
default