This is a repeated dose, open label trial investigating safety, efficacy, PD and PK of FE 203799 in 8 patients with SBS. The patients will receive a subcutaneous (SC) dose of 5 mg FE 203799 once weekly for 4 consecutive weeks, and efficacy parameters and PK will be assessed after the fourth dose. Safety follow up assessments will be performed 4-6 weeks after the last dose.
This is a repeated dose, open label trial investigating safety, efficacy, PD and PK of FE 203799 in 8 patients with SBS . The patients will receive a subcutaneous (SC) dose of 5 mg FE 203799 once weekly for 4 consecutive weeks, and efficacy parameters and PK will be assessed after the fourth dose. Safety follow-up assessments will be performed 4-6 weeks after the last dose. The first two administrations of trial drug will be performed at the clinic, while the third dose can be either self-administered by the patient or administered at the clinic if the patient prefers to travel to the site or other considerations make a site visit preferable. The fourth administration of trial drug will be performed at the clinic just prior to assessment of efficacy parameters in the treatment balance study. Prior to each administration of trial drug, liver function parameters will be analysed and assessed. During the entire trial, patients who develop extremely high or persistently elevated liver enzymes following trial drug administration will be discontinued from the trial. The patients will complete a diary with data on trial drug administration performed at home, local tolerability and adverse events (AEs).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
SUPPORTIVE_CARE
Masking
NONE
Enrollment
8
FE203799 5 mg subcutaneous once weekly
Rigshospitalet
Copenhagen, Denmark
Incidence of treatment-emergent adverse events
Adverse events as assessed by CTCAE v4.03
Time frame: Day -28 to Day 26 plus 6 weeks
Assessment of intestinal insufficiency and gut absorption
Changes in the wet weight (g) of faecal excretion
Time frame: Day -28 to day 26
Assessment of intestinal insufficiency and gut absorption
Changes from baseline in lean body mass by DEXA scan.
Time frame: Day -28 and day 26
Assessment of intestinal insufficiency and gut absorption
Changes from baseline in bone mineral content by DEXA scan.
Time frame: Day -28 and day 26
Assessment of intestinal insufficiency and gut absorption
Changes from baseline in fat mass by DEXA scan.
Time frame: Day -28 and day 26
Assessment of intestinal insufficiency and gut absorption
Measure of body weight (kg)
Time frame: Day -28 to day 26 plus 6 weeks
Assessment of intestinal insufficiency and gut absorption
Measurement of urinary output (ml)
Time frame: Baseline (Day -6) to end of treatment (Day 26)
Assessment of intestinal insufficiency and gut absorption
Measurement of urinary electrolytes (sodium, potassium, calcium, magnesium) (mmol/d)
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Time frame: Baseline (Day -6) to end of treatment (Day 26)
Assessment of gut regeneration
Measurements of the plasma Citrulline (ng/ml)
Time frame: Baseline (Day -7) to end of treatment period (Day 26) plus 6 weeks
Maximum observed plasma concentration (Cmax)
Cmax (ng/ml)
Time frame: Day 1 to end of treatment period (Day 26)
Cumulative area under the plasma concentration (AUC) of FE 203799 from time 0 to 168
AUC0-168 (ng\*hr/ml)
Time frame: Day 1 to end of treatment period (Day 26)
Terminal elimination half-life (T1/2) of FE 203799
T1/2 (h)
Time frame: Day 1 to end of treatment period (Day 26)
Apparent total body clearance (CL) of FE 203799 from plasma
CL (ml/hr)
Time frame: Day 1 to end of treatment period (Day 26)
Apparent volume of distribution (Vd) of FE 203799 during terminal phase
Vd (ml)
Time frame: Day 1 to end of treatment period (Day 26)