Trial to Evaluate Efficacy and Safety of Apraglutide in SBS-IF

Phase 3

Completed

Conditions: Short Bowel Syndrome

Interventions: Drug: apraglutide

Registration Number: NCT04627025

Lead Sponsor: VectivBio AG

Brief Summary: 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.

Detailed Description: 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 164

Inclusion Criteria

Not provided

Exclusion Criteria

Pregnancy or lactation.
Major abdominal surgery in the last 6 months prior to screening.
History of cancer (including colon carcinoma) or clinically significant lymphoproliferative disease within ≤5 years, except for adequately treated basal cell skin cancer.
Evidence of active inflammatory GI conditions in the previous 6 months.
Evidence of decompensated heart failure.
Evidence of severe renal or hepatic impairment.
Any previous use of growth factors such as growth hormone (GH), native GLP-2, GLP-1, or GLP-2 or GLP-1 analogues should be discussed with the Investigator.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Apraglutide SC injections, once weekly	apraglutide	Peptide analogue of GLP-2
Placebo	apraglutide	Placebo for apraglutide, SC injection once weekly

Primary Outcome Measures

Name	Time	Method
Relative change from baseline in actual weekly PS volume at Week 24.	At week 24 of treatment

Secondary Outcome Measures

Name	Time	Method
Subjects who achieve a reduction of at least 1 day per week of PS at Weeks 24 / 48 (CIC only).	Weeks 24 and 48 of treatment
Relative change from baseline in actual weekly PS volume at Weeks 12 / 24 / 48 (CIC only).	Weeks 12 / 24 / 48 of treatment
Subjects reaching enteral autonomy at Weeks 24 / 48 (CIC only).	Weeks 24 / 48 of treatment
At least 20% reduction of PS volume from baseline at Weeks 20 / 24.	Weeks 20 / 24 of treatment
Calorie reduction in the PN at Weeks 24.	Weeks 24 of treatment
Change from baseline on the Pittsburgh Sleep Quality Inventory (PSQI) at Weeks 24 / 48 (CIC only)	Weeks 24 / 48 of treatment
Change from baseline on the Patient Global Impression of Change (PGIC) at Weeks 24 / 48 (CIC only)	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).	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).	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).	Weeks 24 / 48 of treatment
Absorption rate constant (ka) of apraglutide through population PK data analysis	Weeks 0 / 24 of treatment
Apparent clearance (CL/F) of apraglutide through population PK data analysis	Weeks 0 / 24 of treatment
Apparent volume of distribution (Vz/F) of apraglutide through population PK data analysis	Weeks 0 / 24 of treatment
At least 40% reduction of PS volume at Weeks 20 and 24 (Stoma only)	Weeks 20 / 24 of treatment
Trough plasma concentrations (Ctrough) at every other visit during the prolonged dosing period (CIC only)	Weeks 24 / 48 of treatment

Trial Locations

Locations (68): 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
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Ronald Reagan UCLA Medical Center
🇺🇸Los Angeles, California, United States