The primary objective of the trial is the confirmation of the efficacy of apraglutide to evaluate the efficacy of weekly subcutaneous apraglutide in reducing parenteral support dependency.
This is an international, multicenter, double-blind, randomized, placebo-controlled, trial to evaluate the efficacy and safety of weekly SC injections of apraglutide in adult subjects with SBS-IF. The active pharmaceutical ingredient is apraglutide, a GLP-2 analogue. The trial consists of a screening phase, a treatment phase of 48 weeks, and a safety follow-up phase. Trial participants will receive, after successful screening, once weekly apraglutide.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
164
Apraglutide is a peptide analogue of GLP-2 under development for treatment of SBS-IF, which acts as a full agonist at the GLP-2 receptor with in vitro potency and selectivity comparable with native GLP-2.
Relative change from baseline in actual weekly PS volume at Week 24.
Time frame: At week 24 of treatment
Subjects who achieve a reduction of at least 1 day per week of PS at Weeks 24 / 48 (CIC only).
Time frame: Weeks 24 and 48 of treatment
Relative change from baseline in actual weekly PS volume at Weeks 12 / 24 / 48 (CIC only).
Time frame: Weeks 12 / 24 / 48 of treatment
Subjects reaching enteral autonomy at Weeks 24 / 48 (CIC only).
Time frame: Weeks 24 / 48 of treatment
At least 20% reduction of PS volume from baseline at Weeks 20 / 24.
Time frame: Weeks 20 / 24 of treatment
Calorie reduction in the PN at Weeks 24.
Time frame: Weeks 24 of treatment
Change from baseline on the Pittsburgh Sleep Quality Inventory (PSQI) at Weeks 24 / 48 (CIC only)
Time frame: Weeks 24 / 48 of treatment
Change from baseline on the Patient Global Impression of Change (PGIC) at Weeks 24 / 48 (CIC only)
Time frame: Weeks 24 / 48 of treatment.
Change from baseline on the Patient Global Impression of Treatment Satisfaction (PGI-TS) at week 24 and 48 (CIC only).
Time frame: Weeks 24 / 48 of treatment
Change from baseline on the Patient Global Impression of Satisfaction with Parenteral Support (PGI-SPS) at Week 24 and 48 (CIC only).
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Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
Gastroenterology Group of Naples
Naples, Florida, United States
Northwestern University
Chicago, Illinois, United States
Henry Ford Medical Center - Columbus
Novi, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Mount Sinai Medical Center
New York, New York, United States
Duke University Hospital
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
...and 58 more locations
Time frame: Weeks 24 / 48 of treatment
Change from baseline on the Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and 48 (CIC only).
Time frame: Weeks 24 / 48 of treatment
Absorption rate constant (ka) of apraglutide through population PK data analysis
Time frame: Weeks 0 / 24 of treatment
Apparent clearance (CL/F) of apraglutide through population PK data analysis
Time frame: Weeks 0 / 24 of treatment
Apparent volume of distribution (Vz/F) of apraglutide through population PK data analysis
Time frame: Weeks 0 / 24 of treatment
At least 40% reduction of PS volume at Weeks 20 and 24 (Stoma only)
Time frame: Weeks 20 / 24 of treatment
Trough plasma concentrations (Ctrough) at every other visit during the prolonged dosing period (CIC only)
Time frame: Weeks 24 / 48 of treatment